Author: "Montini E" - Searchworks@Jio Institute Digital Library Search Results

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338 results on '"Montini E"'

1. Normalization of clonal diversity in gene therapy studies using shape constrained splines

Author: Del Core, L., Cesana, D., Gallina, P., Secanechia, Y. N. Serina, Rudilosso, L., Montini, E., Wit, E. C., Calabria, A., and Grzegorczyk, M. A.
Published: 2022
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2. Empirical evidence from the design of a MaaS platform

Author: Corti, D., Bettoni, A., Montini, E., Barni, A., and Arica, E.
Published: 2021
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3. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

Author: Scala, S, Ferrua, F, Basso-Ricci, L, Dionisio, F, Omrani, M, Quaranta, P, Jofra Hernandez, R, Del Core, L, Benedicenti, F, Monti, I, Giannelli, S, Fraschetta, F, Darin, S, Albertazzi, E, Galimberti, S, Montini, E, Calabria, A, Cicalese, M, Aiuti, A, Scala, Serena, Ferrua, Francesca, Basso-Ricci, Luca, Dionisio, Francesca, Omrani, Maryam, Quaranta, Pamela, Jofra Hernandez, Raisa, Del Core, Luca, Benedicenti, Fabrizio, Monti, Ilaria, Giannelli, Stefania, Fraschetta, Federico, Darin, Silvia, Albertazzi, Elena, Galimberti, Stefania, Montini, Eugenio, Calabria, Andrea, Cicalese, Maria Pia, Aiuti, Alessandro, Scala, S, Ferrua, F, Basso-Ricci, L, Dionisio, F, Omrani, M, Quaranta, P, Jofra Hernandez, R, Del Core, L, Benedicenti, F, Monti, I, Giannelli, S, Fraschetta, F, Darin, S, Albertazzi, E, Galimberti, S, Montini, E, Calabria, A, Cicalese, M, Aiuti, A, Scala, Serena, Ferrua, Francesca, Basso-Ricci, Luca, Dionisio, Francesca, Omrani, Maryam, Quaranta, Pamela, Jofra Hernandez, Raisa, Del Core, Luca, Benedicenti, Fabrizio, Monti, Ilaria, Giannelli, Stefania, Fraschetta, Federico, Darin, Silvia, Albertazzi, Elena, Galimberti, Stefania, Montini, Eugenio, Calabria, Andrea, Cicalese, Maria Pia, and Aiuti, Alessandro
Abstract: Mobilized peripheral blood is increasingly used instead of bone marrow as a source of autologous hematopoietic stem/progenitor cells for ex vivo gene therapy. Here, we present an unplanned exploratory analysis evaluating the hematopoietic reconstitution kinetics, engraftment and clonality in 13 pediatric Wiskott-Aldrich syndrome patients treated with autologous lentiviral-vector transduced hematopoietic stem/progenitor cells derived from mobilized peripheral blood (n = 7), bone marrow (n = 5) or the combination of the two sources (n = 1). 8 out of 13 gene therapy patients were enrolled in an open-label, non-randomized, phase 1/2 clinical study (NCT01515462) and the remaining 5 patients were treated under expanded access programs. Although mobilized peripheral blood- and bone marrow- hematopoietic stem/progenitor cells display similar capability of being gene-corrected, maintaining the engineered grafts up to 3 years after gene therapy, mobilized peripheral blood-gene therapy group shows faster neutrophil and platelet recovery, higher number of engrafted clones and increased gene correction in the myeloid lineage which correlate with higher amount of primitive and myeloid progenitors contained in hematopoietic stem/progenitor cells derived from mobilized peripheral blood. In vitro differentiation and transplantation studies in mice confirm that primitive hematopoietic stem/progenitor cells from both sources have comparable engraftment and multilineage differentiation potential. Altogether, our analyses reveal that the differential behavior after gene therapy of hematopoietic stem/progenitor cells derived from either bone marrow or mobilized peripheral blood is mainly due to the distinct cell composition rather than functional differences of the infused cell products, providing new frames of references for clinical interpretation of hematopoietic stem/progenitor cell transplantation outcome.
Published: 2023

4. Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition

Author: Zaghi, M, Banfi, F, Massimino, L, Volpin, M, Bellini, E, Brusco, S, Merelli, I, Barone, C, Bruni, M, Bossini, L, Lamparelli, L, Pintado, L, D'Aliberti, D, Spinelli, S, Mologni, L, Colasante, G, Ungaro, F, Cioni, J, Azzoni, E, Piazza, R, Montini, E, Broccoli, V, Sessa, A, Zaghi, Mattia, Banfi, Federica, Massimino, Luca, Volpin, Monica, Bellini, Edoardo, Brusco, Simone, Merelli, Ivan, Barone, Cristiana, Bruni, Michela, Bossini, Linda, Lamparelli, Luigi Antonio, Pintado, Laura, D'Aliberti, Deborah, Spinelli, Silvia, Mologni, Luca, Colasante, Gaia, Ungaro, Federica, Cioni, Jean-Michel, Azzoni, Emanuele, Piazza, Rocco, Montini, Eugenio, Broccoli, Vania, Sessa, Alessandro, Zaghi, M, Banfi, F, Massimino, L, Volpin, M, Bellini, E, Brusco, S, Merelli, I, Barone, C, Bruni, M, Bossini, L, Lamparelli, L, Pintado, L, D'Aliberti, D, Spinelli, S, Mologni, L, Colasante, G, Ungaro, F, Cioni, J, Azzoni, E, Piazza, R, Montini, E, Broccoli, V, Sessa, A, Zaghi, Mattia, Banfi, Federica, Massimino, Luca, Volpin, Monica, Bellini, Edoardo, Brusco, Simone, Merelli, Ivan, Barone, Cristiana, Bruni, Michela, Bossini, Linda, Lamparelli, Luigi Antonio, Pintado, Laura, D'Aliberti, Deborah, Spinelli, Silvia, Mologni, Luca, Colasante, Gaia, Ungaro, Federica, Cioni, Jean-Michel, Azzoni, Emanuele, Piazza, Rocco, Montini, Eugenio, Broccoli, Vania, and Sessa, Alessandro
Abstract: Within the chromatin, distal elements interact with promoters to regulate specific transcriptional programs. Histone acetylation, interfering with the net charges of the nucleosomes, is a key player in this regulation. Here, we report that the oncoprotein SET is a critical determinant for the levels of histone acetylation within enhancers. We disclose that a condition in which SET is accumulated, the severe Schinzel-Giedion Syndrome (SGS), is characterized by a failure in the usage of the distal regulatory regions typically employed during fate commitment. This is accompanied by the usage of alternative enhancers leading to a massive rewiring of the distal control of the gene transcription. This represents a (mal)adaptive mechanism that, on one side, allows to achieve a certain degree of differentiation, while on the other affects the fine and corrected maturation of the cells. Thus, we propose the differential in cis-regulation as a contributing factor to the pathological basis of SGS and possibly other the SET-related disorders in humans.
Published: 2023

5. A meta-model for modular composition of tailored human digital twins in production

Author: Mourtzis, D, Montini, E, Bettoni, A, Ciavotta, M, Carpanzano, E, Pedrazzoli, P, Montini E., Bettoni A., Ciavotta M., Carpanzano E., Pedrazzoli P., Mourtzis, D, Montini, E, Bettoni, A, Ciavotta, M, Carpanzano, E, Pedrazzoli, P, Montini E., Bettoni A., Ciavotta M., Carpanzano E., and Pedrazzoli P.
Abstract: Multiple and diverse factory digital twins have been proposed in the literature. However, despite the recognized growing importance of workers in smart and autonomous industrial settings, such models still lack or oversimplify human representation. Human digital twins must include human monitoring and behavioural data and models based on psychophysical status, knowledge, skills, and personal needs to manage production systems that aim, at the same time, to achieve process performance and workers' wellbeing. This paper proposes a metamodel based on data, events, and connectors that supports the modular composition of tailored human digital twins. This work also addresses an industrial application of the metamodel for preliminary validation.
Published: 2021

6. ESICM LIVES 2016: part three: Milan, Italy. 1–5 October 2016

Author: Velasquez, T., Mackey, G., Lusk, J., Kyle, U. G., Fontenot, T., Marshall, P., Shekerdemian, L. S., Coss-Bu, J. A., Nishigaki, A., Yatabe, T., Tamura, T., Yamashita, K., Yokoyama, M., Ruiz-Rodriguez, J. C., Encina, B., Belmonte, R., Troncoso, I., Tormos, P., Riveiro, M., Baena, J., Sanchez, A., Bañeras, J., Cordón, J., Duran, N., Ruiz, A., Caballero, J., Nuvials, X., Riera, J., Serra, J., Rutten, A. M. F., van Ieperen, S. N. M., Der Kinderen, E. P. H. M., Van Logten, T., Kovacikova, L., Skrak, P., Zahorec, M., Kyle, U. G., Akcan-Arikan, A., Silva, J. C., Mackey, G., Lusk, J., Goldsworthy, M., Shekerdemian, L. S., Coss-Bu, J. A., Wood, D., Harrison, D., Parslow, R., Davis, P., Pappachan, J., Goodwin, S., Ramnarayan, P., Chernyshuk, S., Yemets, H., Zhovnir, V., Pulitano’, S. M., De Rosa, S., Mancino, A., Villa, G., Tosi, F., Franchi, P., Conti, G., Patel, B., Khine, H., Shah, A., Sung, D., Singer, L., Haghbin, S., Inaloo, S., Serati, Z., Idei, M., Nomura, T., Yamamoto, N., Sakai, Y., Yoshida, T., Matsuda, Y., Yamaguchi, Y., Takaki, S., Yamaguchi, O., Goto, T., Longani, N., Medar, S., Abdel-Aal, I. R., El Adawy, A. S., Mohammed, H. M. E. H., Mohamed, A. N., Parry, S. M., Knight, L. D., Denehy, L., De Morton, N., Baldwin, C. E., Sani, D., Kayambu, G., da Silva, V. Z. M., Phongpagdi, P., Puthucheary, Z. A., Granger, C. L., Rydingsward, J. E., Horkan, C. M., Christopher, K. B., McWilliams, D., Jones, C., Reeves, E., Atkins, G., Snelson, C., Aitken, L. M., Rattray, J., Kenardy, J., Hull, A. M., Ullman, A., Le Brocque, R., Mitchell, M., Davis, C., Macfarlane, B., Azevedo, J. C., Rocha, L. L., De Freitas, F. F. M., Cavalheiro, A. M., Lucinio, N. M., Lobato, M. S., Ebeling, G., Kraegpoeth, A., Laerkner, E., De Brito-Ashurst, I., White, C., Gregory, S., Forni, L. G., Flowers, E., Curtis, A., Wood, C. A., Siu, K., Venkatesan, K., Muhammad, J. B. H., Ng, L., Seet, E., Baptista, N., Escoval, A., Tomas, E., Agrawal, R., Mathew, R., Varma, A., Dima, E., Charitidou, E., Perivolioti, E., Pratikaki, M., Vrettou, C., Giannopoulos, A., Zakynthinos, S., Routsi, C., Atchade, E., Houzé, S., Jean-Baptiste, S., Thabut, G., Genève, C., Tanaka, S., Lortat-Jacob, B., Augustin, P., Desmard, M., Montravers, P., de Molina, F. J. González, Barbadillo, S., Alejandro, R., Álvarez-Lerma, F., Vallés, J., Catalán, R. M., Palencia, E., Jareño, A., Granada, R. M., Ignacio, M. L., Cui, N., Liu, D., Wang, H., Su, L., Qiu, H., Li, R., Jaffal, K., Rouzé, A., Poissy, J., Sendid, B., Nseir, S., Paramythiotou, E., Rizos, M., Frantzeskaki, F., Antoniadou, A., Vourli, S., Zerva, L., Armaganidis, A., Riera, J., Gottlieb, J., Greer, M., Wiesner, O., Martínez, M., Acuña, M., Rello, J., Welte, T., Atchade, E., Mignot, T., Houzé, S., Jean-Baptiste, S., Thabut, G., Lortat-Jacob, B., Tanaka, S., Augustin, P., Desmard, M., Montravers, P., Soussi, S., Dudoignon, E., Ferry, A., Chaussard, M., Benyamina, M., Alanio, A., Touratier, S., Chaouat, M., Lafaurie, M., Mimoun, M., Mebazaa, A., Legrand, M., Sheils, M. A., Patel, C., Mohankumar, L., Akhtar, N., Noriega, S. K. Pacheco, Aldana, N. Navarrete, León, J. L. Ávila, Baquero, J. Durand, Bernal, F. Fernández, Ahmadnia, E., Hadley, J. S., Millar, M., Hall, D., Hewitt, H., Yasuda, H., Sanui, M., Komuro, T., Kawano, S., Andoh, K., Yamamoto, H., Noda, E., Hatakeyama, J., Saitou, N., Okamoto, H., Kobayashi, A., Takei, T., Matsukubo, S., Rotzel, H. B., Lázaro, A. Serrano, Prada, D. Aguillón, Gimillo, M. Rodriguez, Barinas, O. Diaz, Cortes, M. L. Blasco, Franco, J. Ferreres, Roca, J. M. Segura, Carratalá, A., Gonçalves, B., Turon, R., Mendes, A., Miranda, F., Mata, P. J., Cavalcanti, D., Melo, N., Lacerda, P., Kurtz, P., Righy, C., Rosario, L. E. de la Cruz, Lesmes, S. P. Gómez, Romero, J. C. García, Herrera, A. N. García, Pertuz, E. D. Díaz, Sánchez, M. J. Gómez, Sanz, E. Regidor, Hualde, J. Barado, Hernández, A. Ansotegui, Irazabal, J. M. Guergué, Spatenkova, V., Bradac, O., Suchomel, P., Urli, T., Lazzeri, E. Heusch, Aspide, R., Zanello, M., Perez-Borrero, L., Garcia-Alvarez, J. M., Arias-Verdu, M. D., Aguilar-Alonso, E., Rivera-Fernandez, R., Mora-Ordoñez, J., De La Fuente-Martos, C., Castillo-Lorente, E., Guerrero-Lopez, F., Lesmes, S. P. Gómez, Rosario, L. E. De la Cruz, Pertuz, E. D. Díaz, Hernández, A. Ansotegui, Romero, J. C. García, Sánchez, M. J. Gómez, Herrera, A. N. García, Ramírez, J. Roldán, Sanz, E. Regidor, Hualde, J. Barado, León, J. P. Tirapu, Navarro-Guillamón, L., Cordovilla-Guardia, S., Iglesias-Santiago, A., Guerrero-López, F., Fernández-Mondéjar, E., Vidal, A., Perez, M., Juez, A., Arias, N., Colino, L., Perez, J. L., Pérez, H., Calpe, P., Alcala, M. A., Robaglia, D., Perez, C., Lan, S. K., Cunha, M. M., Moreira, T., Santos, F., Lafuente, E., Fernandes, M. J., Silva, J. G., Rosario, L. E. de la Cruz, Lesmes, S. P. Gómez, Herrera, A. N. García, Romero, J. C. García, Pertuz, E. D. Díaz, Sánchez, M. J. Gómez, Sanz, E. Regidor, Echeverría, J. G. Armando, Hernández, A. Ansotegui, Hualde, J. Barado, Podlepich, V., Sokolova, E., Alexandrova, E., Lapteva, K., Kurtz, P., Shuinotsuka, C., Rabello, L., Vianna, G., Reis, A., Cairus, C., Salluh, J., Bozza, F., Torres, J. C. Barrios, Araujo, N. J. Fernández, García-Olivares, P., Keough, E., Dalorzo, M., Tang, L. K., De Sousa, I., Díaz, M., Marcos-Zambrano, L. J., Guerrero, J. E., Gomez, S. E. Zamora, Lopez, G. D. Hernandez, Cuellar, A. I. Vazquez, Nieto, O. R. Perez, Gonzalez, J. A. Castanon, Bhasin, D., Rai, S., Singh, H., Gupta, O., Bhattal, M. K., Sampley, S., Sekhri, K., Nandha, R., Aliaga, F. A., Olivares, F., Appiani, F., Farias, P., Alberto, F., Hernández, A., Pons, S., Sonneville, R., Bouadma, L., Neuville, M., Mariotte, E., Radjou, A., Lebut, J., Chemam, S., Voiriot, G., Dilly, M. P., Mourvillier, B., Dorent, R., Nataf, P., Wolff, M., Timsit, J. F., Ediboglu, O., Ataman, S., Ozkarakas, H., Kirakli, C., Vakalos, A., Avramidis, V., Obukhova, O., Kurmukov, I. A., Kashiya, S., Golovnya, E., Baikova, V. N., Ageeva, T., Haritydi, T., Kulaga, E. V., Rios-Toro, J. J., Perez-Borrero, L., Aguilar-Alonso, E., Arias-Verdu, M. D., Garcia-Alvarez, J. M., Lopez-Caler, C., De La Fuente-Martos, C., Rodriguez-Fernandez, S., Sanchez-Orézzoli, M. Gomez, Martin-Gallardo, F., Nikhilesh, J., Joshi, V., Villarreal, E., Ruiz, J., Gordon, M., Quinza, A., Gimenez, J., Piñol, M., Castellanos, A., Ramirez, P., Jeon, Y. D., Jeong, W. Y., Kim, M. H., Jeong, I. Y., Ahn, M. Y., Ahn, J. Y., Han, S. H., Choi, J. Y., Song, Y. G., Kim, J. M., Ku, N. S., Shah, H., Kellner, F., Rezai, F., Mistry, N., Yodice, P., Ovnanian, V., Fless, K., Handler, E., Alejos, R. Martínez, Romeu, J. D. Martí, Antón, D. González, Quinart, A., Martí, A. Torres, Llaurado-Serra, M., Lobo-Civico, A., Ventura-Rosado, A., Piñol-Tena, A., Pi-Guerrero, M., Paños-Espinosa, C., Peralvo-Bernat, M., Marine-Vidal, J., Gonzalez-Engroba, R., Montesinos-Cerro, N., Treso-Geira, M., Valeiras-Valero, A., Martinez-Reyes, L., Sandiumenge, A., Jimenez-Herrera, M. F., Helyar, S., Riozzi, P., Noon, A., Hallows, G., Cotton, H., Keep, J., Hopkins, P. A., Taggu, A., Renuka, S., Sampath, S., Rood, P. J. T., Frenzel, T., Verhage, R., Bonn, M., Pickkers, P., van der Hoeven, J. G., van den Boogaard, M., Corradi, F., Melnyk, L., Moggia, F., Pienovi, R., Adriano, G., Brusasco, C., Mariotti, L., Lattuada, M., Bloomer, M. J., Coombs, M., Ranse, K., Endacott, R., Maertens, B., Blot, K., Blot, S., Amerongen, M. P. van Nieuw, van der Heiden, E. S., Twisk, J. W. R., Girbes, A. R. J., Spijkstra, J. J., Riozzi, P., Helyar, S., Cotton, H., Hallows, G., Noon, A., Bell, C., Peters, K., Feehan, A., Keep, J., Hopkins, P. A., Churchill, K., Hawkins, K., Brook, R., Paver, N., Endacott, R., Maistry, N., van Wijk, A., Rouw, N., van Galen, T., Evelein-Brugman, S., Taggu, A., Krishna, B., Sampath, S., Putzu, A., Fang, M., Berto, M. Boscolo, Belletti, A., Cassina, T., Cabrini, L., Mistry, M., Alhamdi, Y., Welters, I., Abrams, S. T., Toh, C. H., Han, H. S., Gil, E. M., Lee, D. S., Park, C. M., Winder-Rhodes, S., Lotay, R., Doyle, J., Ke, M. W., Huang, W. C., Chiang, C. H., Hung, W. T., Cheng, C. C., Lin, K. C., Lin, S. C., Chiou, K. R., Wann, S. R., Shu, C. W., Kang, P. L., Mar, G. Y., Liu, C. P., Dubó, S., Aquevedo, A., Jibaja, M., Berrutti, D., Labra, C., Lagos, R., García, M. F., Ramirez, V., Tobar, M., Picoita, F., Peláez, C., Carpio, D., Alegría, L., Hidalgo, C., Godoy, K., Bakker, J., Hernández, G., Sadamoto, Y., Katabami, K., Wada, T., Ono, Y., Maekawa, K., Hayakawa, M., Sawamura, A., Gando, S., Marin-Mateos, H., Perez-Vela, J. L., Garcia-Gigorro, R., Peiretti, M. A. Corres, Lopez-Gude, M. J., Chacon-Alves, S., Renes-Carreño, E., Montejo-González, J. C., Parlevliet, K. L., Touw, H. R. W., Beerepoot, M., Boer, C., Elbers, P. W. G., Tuinman, P. R., Abdelmonem, S. A., Helmy, T. A., El Sayed, I., Ghazal, S., Akhlagh, S. H., Masjedi, M., Hozhabri, K., Kamali, E., Zýková, I., Paldusová, B., Sedlák, P., Morman, D., Youn, A. M., Ohta, Y., Sakuma, M., Bates, D., Morimoto, T., Su, P. L., Chang, W. Y., Lin, W. C., Chen, C. W., Facchin, F., Zarantonello, F., Panciera, G., De Cassai, A., Venrdramin, A., Ballin, A., Tonetti, T., Persona, P., Ori, C., Del Sorbo, L., Rossi, S., Vergani, G., Cressoni, M., Chiumello, D., Chiurazzi, C., Brioni, M., Algieri, I., Tonetti, T., Guanziroli, M., Colombo, A., Tomic, I., Colombo, A., Crimella, F., Carlesso, E., Gasparovic, V., Gattinoni, L., Neto, A. Serpa, Schmidt, M., Pham, T., Combes, A., de Abreu, M. Gama, Pelosi, P., Schultz, M. J., Katira, B. H., Engelberts, D., Giesinger, R. E., Ackerley, C., Yoshida, T., Zabini, D., Otulakowski, G., Post, M., Kuebler, W. M., McNamara, P. J., Kavanagh, B. P., Pirracchio, R., Rigon, M. Resche, Carone, M., Chevret, S., Annane, D., Eladawy, S., El-Hamamsy, M., Bazan, N., Elgendy, M., De Pascale, G., Vallecoccia, M. S., Cutuli, S. L., Di Gravio, V., Pennisi, M. A., Conti, G., Antonelli, M., Andreis, D. T., Khaliq, W., Singer, M., Hartmann, J., Harm, S., Carmona, S. Alcantara, Almudevar, P. Matia, Abellán, A. Naharro, Ramos, J. Veganzones, Pérez, L. Pérez, Valbuena, B. Lobo, Sanz, N. Martínez, Simón, I. Fernández, Arrigo, M., Feliot, E., Deye, N., Cariou, A., Guidet, B., Jaber, S., Leone, M., Resche-Rigon, M., Baron, A. Vieillard, Legrand, M., Gayat, E., Mebazaa, A., Balik, M., Kolnikova, I., Maly, M., Waldauf, P., Tavazzi, G., Kristof, J., Herpain, A., Su, F., Post, E., Taccone, F., Vincent, J. L., Creteur, J., Lee, C., Hatib, F., Jian, Z., Buddi, S., Cannesson, M., Fileković, S., Turel, M., Knafelj, R., Gorjup, V., Stanić, R., Gradišek, P., Cerović, O., Mirković, T., Noč, M., Tirkkonen, J., Hellevuo, H., Olkkola, K. T., Hoppu, S., Lin, K. C., Hung, W. T., Chiang, C. C., Huang, W. C., Juan, W. C., Lin, S. C., Cheng, C. C., Lin, P. H., Fong, K. Y., Hou, D. S., Kang, P. L., Wann, S. R., Chen, Y. S., Mar, G. Y., Liu, C. P., Paul, M., Bougouin, W., Geri, G., Dumas, F., Champigneulle, B., Legriel, S., Charpentier, J., Mira, J. P., Sandroni, C., Cariou, A., Zimmerman, J., Sullivan, E., Noursadeghi, M., Fox, B., Sampson, D., McHugh, L., Yager, T., Cermelli, S., Seldon, T., Bhide, S., Brandon, R. A., Brandon, R. B., Zwaag, J., Beunders, R., Pickkers, P., Kox, M., Gul, F., Arslantas, M. K., Genc, D., Zibandah, N., Topcu, L., Akkoc, T., Cinel, I., Greco, E., Lauretta, M. P., Andreis, D. T., Singer, M., Garcia, I. Palacios, Cordero, M., Martin, A. Diaz, Pallás, T. Aldabó, Montero, J. Garnacho, Rey, J. Revuelto, Malo, L. Roman, Montoya, A. A. Tanaka, Martinez, A. D. C. Amador, Ayala, L. Y. Delgado, Zepeda, E. Monares, Granillo, J. Franco, Sanchez, J. Aguirre, Alejo, G. Camarena, Cabrera, A. Rugerio, Montenegro, A. Pedraza, Pham, T., Beduneau, G., Schortgen, F., Piquilloud, L., Zogheib, E., Jonas, M., Grelon, F., Runge, I., Terzi, N., Grangé, S., Barberet, G., Guitard, P. G., Frat, J. P., Constan, A., Chrétien, J. M., Mancebo, J., Mercat, A., Richard, J. C. M., Brochard, L., Soilemezi, E., Koco, E., Savvidou, S., Nouris, C., Matamis, D., Di Mussi, R., Spadaro, S., Volta, C. A., Mariani, M., Colaprico, A., Antonio, C., Bruno, F., Grasso, S., Rodriguez, A., Martín-Loeches, I., Díaz, E., Masclans, J. R., Gordo, F., Solé-Violán, J., Bodí, M., Avilés-Jurado, F. X., Trefler, S., Magret, M., Reyes, L. F., Marín-Corral, J., Yebenes, J. C., Esteban, A., Anzueto, A., Aliberti, S., Restrepo, M. I., Larsson, J. Skytte, Redfors, B., Ricksten, S. 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S., Kashyap, R., Gajic, O., Yoshihiro, S., Sakuraya, M., Hayakawa, M., Hirata, A., Kawamura, N., Tsutui, T., Yoshida, K., Hashimoto, Y., Chang, C. H., Hu, H. C., Chiu, L. C., Hung, C. Y., Li, S. H., Kao, K. C., Sibley, S., Drover, J., D’Arsigny, C., Parker, C., Howes, D., Moffatt, S., Erb, J., Ilan, R., Messenger, D., Ball, I., Boyd, J. G., Harrison, M., Ridi, S., Muscedere, J., Andrade, A. H., Costa, R. C., Souza, V. A., Gonzalez, V., Amorim, V., Rolla, F., Filho, C. A. C. Abreu, Miranda, R., Atchasiri, S., Buranavanich, P., Wathanawatthu, T., Suwanpasu, S., Bureau, C., Rolland-Debord, C., Poitou, T., Clavel, M., Perbet, S., Terzi, N., Kouatchet, A., Similowski, T., Demoule, A., Diaz, P., Nunes, J., Escórcio, S., Silva, G., Chaves, S., Jardim, M., Câmara, M., Fernandes, N., Duarte, R., Jardim, J. J., Pereira, C. A., Nóbrega, J. J., Chen, C. M., Lai, C. C., Cheng, K. C., Chou, W., Lee, S. J., Cha, Y. S., Lee, W. 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Iglesias, Sáez, V. Chica, Ruiz-Ruano, R. de la Chica, González, A. Sánchez, Kunze-Szikszay, N., Wand, S., Klapsing, P., Wetz, A., Heyne, T., Schwerdtfeger, K., Troeltzsch, M., Bauer, M., Quintel, M., Moerer, O., Cook, D. J., Rutherford, W. B., Scales, D. C., Adhikari, N. K., Cuthbertson, B. H., Suzuki, T., Takei, T., Fushimi, K., Iwamoto, M., Nakagawa, S., Mendsaikhan, N., Begzjav, T., Lundeg, G., Dünser, M. W., Romero, D. González, Cabrera, J. L. Santana, Santana, J. D. Martín, Padilla, Y. Santana, Pérez, H. Rodríguez, Torrent, R. Lorenzo, Kleinpell, R., Chouris, I., Radu, V., Stougianni, M., Lavrentieva, A., Lagonidis, D., Price, R. D. T., Day, A., Arora, N., Henderson, M. A., Hickey, S., Costa, M. I. Almeida, Carvalho, J. P., Gomes, A. A., Mergulhão, P. J., Chan, K. K. C., Shum, H. P., Yan, W. W., Maghsoudi, B., Tabei, S. H., Masjedi, M., Sabetian, G., Tabatabaei, H. R., Akbarzadeh, A., Saigal, S., Pakhare, A., Joshi, R., Pattnaik, S. K., Ray, B., Rousseau, A. F., Michel, L., Bawin, M., Cavalier, E., Reginster, J. Y., Damas, P., Bruyere, O., Zhou, J. C., Cauwenberghs, H., De Backer, A., Neels, H., Deblier, I., Berghmans, J., Himpe, D., Barea-Mendoza, J. A., Portillo, I. Prieto, Fernández, M. Valiente, Gigorro, R. Garcia, Vela, J. L. Perez, Mateos, H. Marín, Alves, S. Chacón, Varas, G. Morales, Rodriguez-Biendicho, A., Carreño, E. Renes, González, J. C. Montejo, Yang, J. S., Chiang, C. H., Hung, W. T., Huang, W. C., Cheng, C. C., Lin, K. C., Lin, S. C., Chiou, K. R., Wann, S. R., Lin, K. L., Kang, P. L., Mar, G. Y., Liu, C. P., Zhou, J. C., Choi, Y. J., Yoon, S. Z., Gordillo-Brenes, A., Fernandez-Zamora, M. D., Perez-Borrero, L., Arias-Verdu, M. D., Aguilar-Alonso, E., Herruzo-Aviles, A., Garcia-Delgado, M., Hinojosa-Perez, R., Curiel-Balsera, E., Rivera-Fernandez, R., Lesmes, S. P. Gómez, Rosario, L. E. De la Cruz, Hernández, A. Ansotegui, Herrera, A. N. García, Sanz, E. Regidor, Sánchez, M. J. Gómez, Hualde, J. Barado, Pascual, O. Agudo, León, J. P. Tirapu, Irazabal, J. M. Guergue, Pérez, A. González, Fernández, P. Alvarez, Amor, L. Lopéz, Albaiceta, G. Muñiz, Lesmes, S. P. Gómez, Rosario, L. E. De la Cruz, Hernández, A. Ansotegui, Sanz, E. Regidor, Sánchez, M. J. Gómez, Calvo, S. Aldunate, Herrera, A. N. García, Hualde, J. Barado, Pascual, O. Agudo, León, J. P. Tirapu, Corona, A., Ruffini, C., Spazzadeschi, A., Marrazzo, F., Gandola, A., Sciurti, R., Savi, C., Catena, E., Ke, M. W., Cheng, C. C., Huang, W. C., Chiang, C. H., Hung, W. T., Lin, K. C., Lin, S. C., Wann, S. R., Chiou, K. R., Tseng, C. J., Kang, P. L., Mar, G. Y., Liu, C. P., Bertini, P., De Sanctis, F., Guarracino, F., Bertini, P., Baldassarri, R., Guarracino, F., Buitinck, S. H., van der Voort, P. H. J., Oto, J., Nakataki, E., Tsunano, Y., Izawa, M., Tane, N., Onodera, M., Nishimura, M., Ghosh, S., Gupta, A., De Gasperi, A., Mazza, E., Limuti, R., Prosperi, M., Bissenova, N., Yergaliyeva, A., Talan, L., Yılmaz, G., Güven, G., Yoruk, F., Altıntas, N. D., Mukherjee, D. N., Agarwal, L. K., Mandal, K., Palomar, M., Balsera, B., Vallverdu, M., Martinez, M., Garcia, M., Castellana, D., Lopez, R., Barcenilla, F., Kaminsky, G. E., Carreño, R., Escribá, A., Fuentes, M., Gálvez, V., Del Olmo, R., Nieto, B., Vaquerizo, C., Alvarez, J., De la Torre, M. A., Torres, E., Bogossian, E., Nouer, S. Aranha, Salgado, D. Ribeiro, Brugger, S. Carvalho, Jiménez, G. Jiménez, Torner, M. Miralbés, Vidal, M. Vallverdú, Garrido, B. Balsera, Casals, X. Nuvials, Gaite, F. Barcenilla, Cabello, J. Trujillano, Martínez, M. Palomar, Doganci, M., Izdes, S., Besevli, S. Guzeldag, Alkan, A., Kayaaslan, B., Ramírez, C. Sánchez, Balcázar, L. Caipe, Santana, M. Cabrera, Viera, M. A. Hernández, Escalada, S. Hípola, Vázquez, C. F. Lübbe, Penichet, S. M. Marrero, Campelo, F. Artiles, López, M. A. De La Cal, Santana, P. Saavedra, Santana, S. Ruíz, Repessé, X., Artiguenave, M., Paktoris-Papine, S., Espinasse, F., Dinh, A., El Sayed, F., Charron, C., Géri, G., Vieillard-Baron, A., Marmanidou, K., Oikonomou, M., Nouris, C., Dimitroulakis, K., Soilemezi, E., Matamis, D., Ferré, A., Guillot, M., Teboul, J. L., Lichtenstein, D., Mézière, G., Richard, C., Monnet, X., Pham, T., Beduneau, G., Schortgen, F., Piquilloud, L., Zogheib, E., Jonas, M., Grelon, F., Runge, I., Terzi, N., Grangé, S., Barberet, G., Guitard, P. G., Frat, J. P., Constan, A., Chrétien, J. M., Mancebo, J., Mercat, A., Richard, J. C. M., Brochard, L., Prīdāne, S., Sabeļņikovs, O., Mojoli, F., Orlando, A., Bianchi, I., Torriglia, F., Bianzina, S., Pozzi, M., Iotti, G. A., Braschi, A., Beduneau, G., Pham, T., Schortgen, F., Piquilloud, L., Zogheib, E., Jonas, M., Grelon, F., Runge, I., Terzi, N., Grangé, S., Barberet, G., Guitard, P. G., Frat, J. P., Constan, A., Chrétien, J. M., Mancebo, J., Mercat, A., Richard, J. C. M., Brochard, L., Kondili, E., Psarologakis, C., Kokkini, S., Amargianitakis, V., Babalis, D., Chytas, A., Chouvarda, I., Vaporidi, K., Georgopoulos, D., Trapp, O., Kalenka, A., Mojoli, F., Orlando, A., Bianchi, I., Torriglia, F., Bianzina, S., Pozzi, M., Iotti, G. A., Braschi, A., Lozano, J. A. Benítez, Sánchez, P. Carmona, Francioni, J. E. Barrueco, Ferrón, F. Ruiz, Simón, J. M. Serrano, Spadaro, S., Karbing, D. S., Gioia, A., Moro, F., Corte, F. Dalla, Mauri, T., Volta, C. A., Rees, S. E., Petrova, M. V., Mohan, R., Butrov, A. V., Beeharry, S. D., Vatsik, M. V., Sakieva, F. I., Gobert, F., Yonis, H., Tapponnier, R., Fernandez, R., Labaune, M. A., Burle, J. F., Barbier, J., Vincent, B., Cleyet, M., Richard, J. C., Guérin, C., Shinotsuka, C. Righy, Creteur, J., Taccone, F. S., Törnblom, S., Nisula, S., Vaara, S., Poukkanen, M., Andersson, S., Pettilä, V., Pesonen, E., Xie, Z., Liao, X., Kang, Y., Zhang, J., Kubota, K., Egi, M., Mizobuchi, S., Hegazy, S., El-Keraie, A., El Sayed, E., El Hamid, M. Abd, Rodrigues, N. J., Pereira, M., Godinho, I., Gameiro, J., Neves, M., Gouveia, J., e Silva, Z. Costa, Lopes, J. A., Mckinlay, J., Kostalas, M., Kooner, G., Dudas, G., Horton, A., Kerr, C., Karanjia, N., Creagh-Brown, B., Forni, L., Yamazaki, A., Ganuza, M. Sanz, Molina, J. A. Martinez, Martinez, F. Hidalgo, Freile, M. T. Chiquito, Fernandez, N. Garcia, Travieso, P. Medrano, Bandert, A., Frithiof, R., Lipcsey, M., Smekal, D., Schlaepfer, P., Durovray, J. D., Plouhinec, V., Chiappa, C., Bellomo, R., Schneider, A. G., Mitchell, S., Durrant, J., Street, H., Dunthorne, E., Shears, J., Caballero, C. Hernandez, Hutchison, R., Schwarze, S., Ghabina, S., Thompson, E., Prowle, J. R., Kirwan, C. J., Gonzalez, C. A., Pinto, J. L., Orozco, V., Patiño, J. A., Garcia, P. K., Contreras, K. M., Rodriguez, P., Echeverri, J. E., GETGAG Working Group, JSEPTIC (Japanese Society of Education for Physicians and Trainees in Intensive Care) Clinical Trial Group, CAPCRI Study, for the ReVA Research Network and the PROVE Network Investigators, from the FROG ICU Investigators, The WIND study group, Plug Working Group, GETGAG/SEMICYUC, AKI Research Group, St George’s University of London, IPREA Study Group, FINNRESUSCI Study Group, PICS- HCPA: Programa Intrahospitalar de Combate à Sepse do Hospital de Clínicas de Porto Alegre, ENVIN-HELICS Study Group, ARIAM registry of adult cardiac surgery, The Rapid Diagnosis of Infections in the Critically Ill Team, Tokyo Womens Medical University, PLUG working group, PLUG Working Group, On behalf of Okayama Research Investigation Organizing Network (ORION)investigators, PS-ICU Group, Japan Septic Disseminated Intravascular Coagulation (JSEPTIC DIC) study group, Student Research Committee - Shiraz University of Medical Sciences, ARIAM-ANDALUCIA, The WIND study group, PLUG Working Group, The WIND study group, PLUG Working Group, and Plug working group
Published: 2016
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7. Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting

Author: Cesani, M, Plati, T, Lorioli, L, Benedicenti, F, Redaelli, D, Dionisio, F, Biasco, L, Montini, E, Naldini, L, and Biffi, A
Published: 2015
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8. S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY

Author: Marktel, S, primary, Scaramuzza, S, additional, Giglio, F, additional, Cicalese, M, additional, Lidonnici, M, additional, Rossi, C, additional, Calbi, V, additional, Masera, N, additional, D’Angelo, E, additional, Mirra, N, additional, Origa, R, additional, Tartaglione, I, additional, Perrotta, S, additional, Viarengo, G, additional, Santoleri, L, additional, Milani, R, additional, Gattillo, S, additional, Calabria, A, additional, Montini, E, additional, Graziadei, G, additional, Naldini, L, additional, Cappellini, M, additional, Aiuti, A, additional, Ciceri, F, additional, and Ferrari, G, additional
Published: 2022
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9. In Vitro Isolation of Human Cytotoxic Clones Specific for Autologous Leukemic Blasts

Author: Montagna, D., Aricò, M., Montini, E., De Benedetti, F., Martini, A., Maccario, R., Büchner, T., editor, Hiddemann, W., editor, Wörmann, B., editor, Schellong, G., editor, and Ritter, J., editor
Published: 1994
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10. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

Author: Schiroli, G, Conti, A, Ferrari, S, della Volpe, L, Jacob, A, Albano, L, Beretta, S, Calabria, A, Vavassori, V, Gasparini, P, Salataj, E, Ndiaye-Lobry, D, Brombin, C, Chaumeil, J, Montini, E, Merelli, I, Genovese, P, Naldini, L, Di Micco, R, Schiroli G., Conti A., Ferrari S., della Volpe L., Jacob A., Albano L., Beretta S., Calabria A., Vavassori V., Gasparini P., Salataj E., Ndiaye-Lobry D., Brombin C., Chaumeil J., Montini E., Merelli I., Genovese P., Naldini L., Di Micco R., Schiroli, G, Conti, A, Ferrari, S, della Volpe, L, Jacob, A, Albano, L, Beretta, S, Calabria, A, Vavassori, V, Gasparini, P, Salataj, E, Ndiaye-Lobry, D, Brombin, C, Chaumeil, J, Montini, E, Merelli, I, Genovese, P, Naldini, L, Di Micco, R, Schiroli G., Conti A., Ferrari S., della Volpe L., Jacob A., Albano L., Beretta S., Calabria A., Vavassori V., Gasparini P., Salataj E., Ndiaye-Lobry D., Brombin C., Chaumeil J., Montini E., Merelli I., Genovese P., Naldini L., and Di Micco R.
Abstract: Precise gene editing in hematopoietic stem and progenitor cells (HSPCs) holds promise for treating genetic diseases. However, responses triggered by programmable nucleases in HSPCs are poorly characterized and may negatively impact HSPC engraftment and long-term repopulation capacity. Here, we induced either one or several DNA double-stranded breaks (DSBs) with optimized zinc-finger and CRISPR/Cas9 nucleases and monitored DNA damage response (DDR) foci induction, cell-cycle progression, and transcriptional responses in HSPC subpopulations, with up to single-cell resolution. p53-mediated DDR pathway activation was the predominant response to even single-nuclease-induced DSBs across all HSPC subtypes analyzed. Excess DSB load and/or adeno-associated virus (AAV)-mediated delivery of DNA repair templates induced cumulative p53 pathway activation, constraining proliferation, yield, and engraftment of edited HSPCs. However, functional impairment was reversible when DDR burden was low and could be overcome by transient p53 inhibition. These findings provide molecular and functional evidence for feasible and seamless gene editing in HSPCs. Precise gene editing has the potential to treat immune and hematological diseases. Genovese, Naldini, Di Micco, and colleagues now show that gene-editing procedures are well tolerated by hematopoietic stem cells and provide molecular evidence of the feasibility of seamless gene editing, strengthening translation of such approaches to humans.
Published: 2019

11. The human-digital twin in the manufacturing industry: Current perspectives and a glimpse of future

Author: Montini, E., Bonomi, N., Daniele, F., Bettoni, A., Pedrazzoli, P., Carpanzano, E., and Rocco, P.
Published: 2021

12. Oncogene-induced maladaptive activation of trained immunity in the pathogenesis and treatment of Erdheim-Chester disease.

Author: Molteni, R., Biavasco, R., Stefanoni, D., Nemkov, T., Dominguez Andres, J., Arts, R.J.W., Merelli, I., Mazza, D., Zambrano, S., Panigada, M., Cantoni, E., Tengesdal, I.W., Maksud, P., Piras, F., Cesana, D., Cassina, L., Distefano, G., Loffreda, A., Gnani, D., Luca, G. De, Tomelleri, A., Campochiaro, C., Joosten, L.A.B., Dinarello, C.A., Kajaste-Rudnitski, A., Haroche, J., Cardaci, S., Cenci, S., Dagna, L., Doglioni, C., Ferrarini, M., Ferrero, E., Boletta, A., d'Alessandro, A., Montini, E., Netea, M.G., Cavalli, G.C., Molteni, R., Biavasco, R., Stefanoni, D., Nemkov, T., Dominguez Andres, J., Arts, R.J.W., Merelli, I., Mazza, D., Zambrano, S., Panigada, M., Cantoni, E., Tengesdal, I.W., Maksud, P., Piras, F., Cesana, D., Cassina, L., Distefano, G., Loffreda, A., Gnani, D., Luca, G. De, Tomelleri, A., Campochiaro, C., Joosten, L.A.B., Dinarello, C.A., Kajaste-Rudnitski, A., Haroche, J., Cardaci, S., Cenci, S., Dagna, L., Doglioni, C., Ferrarini, M., Ferrero, E., Boletta, A., d'Alessandro, A., Montini, E., Netea, M.G., and Cavalli, G.C.
Abstract: Item does not contain fulltext, Trained immunity (TI) is a proinflammatory program induced in monocyte/macrophages upon sensing of specific pathogens and is characterized by immunometabolic and epigenetic changes that enhance cytokine production. Maladaptive activation of TI (ie, in the absence of infection) may result in detrimental inflammation and development of disease; however, the exact role and extent of inappropriate activation of TI in the pathogenesis of human diseases is undetermined. In this study, we uncovered the oncogene-induced, maladaptive induction of TI in the pathogenesis of a human inflammatory myeloid neoplasm (Erdheim-Chester disease, [ECD]), characterized by the BRAFV600E oncogenic mutation in monocyte/macrophages and excess cytokine production. Mechanistically, myeloid cells expressing BRAFV600E exhibit all molecular features of TI: activation of the AKT/mammalian target of rapamycin signaling axis; increased glycolysis, glutaminolysis, and cholesterol synthesis; epigenetic changes on promoters of genes encoding cytokines; and enhanced cytokine production leading to hyperinflammatory responses. In patients with ECD, effective therapeutic strategies combat this maladaptive TI phenotype; in addition, pharmacologic inhibition of immunometabolic changes underlying TI (ie, glycolysis) effectively dampens cytokine production by myeloid cells. This study revealed the deleterious potential of inappropriate activation of TI in the pathogenesis of human inflammatory myeloid neoplasms and the opportunity for inhibition of TI in conditions characterized by maladaptive myeloid-driven inflammation.
Published: 2021

13. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

Author: Gentner, B, Tucci, F, Galimberti, S, Fumagalli, F, De Pellegrin, M, Silvani, P, Camesasca, C, Pontesilli, S, Darin, S, Ciotti, F, Sarzana, M, Consiglieri, G, Filisetti, C, Forni, G, Passerini, L, Tomasoni, D, Cesana, D, Calabria, A, Spinozzi, G, Cicalese, M, Calbi, V, Migliavacca, M, Barzaghi, F, Ferrua, F, Gallo, V, Miglietta, S, Zonari, E, Cheruku, P, Forni, C, Facchini, M, Corti, A, Gabaldo, M, Zancan, S, Gasperini, S, Rovelli, A, Boelens, J, Jones, S, Wynn, R, Baldoli, C, Montini, E, Gregori, S, Ciceri, F, Valsecchi, M, la Marca, G, Parini, R, Naldini, L, Aiuti, A, Bernardo, M, Gentner, Bernhard, Tucci, Francesca, Galimberti, Stefania, Fumagalli, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Camesasca, Chiara, Pontesilli, Silvia, Darin, Silvia, Ciotti, Francesca, Sarzana, Marina, Consiglieri, Giulia, Filisetti, Chiara, Forni, Giulia, Passerini, Laura, Tomasoni, Daniela, Cesana, Daniela, Calabria, Andrea, Spinozzi, Giulio, Cicalese, Maria-Pia, Calbi, Valeria, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Gallo, Vera, Miglietta, Simona, Zonari, Erika, Cheruku, Patali S, Forni, Claudia, Facchini, Marcella, Corti, Ambra, Gabaldo, Michela, Zancan, Stefano, Gasperini, Serena, Rovelli, Attilio, Boelens, Jaap-Jan, Jones, Simon A, Wynn, Robert, Baldoli, Cristina, Montini, Eugenio, Gregori, Silvia, Ciceri, Fabio, Valsecchi, Maria G, la Marca, Giancarlo, Parini, Rossella, Naldini, Luigi, Aiuti, Alessandro, Bernardo, Maria-Ester, Gentner, B, Tucci, F, Galimberti, S, Fumagalli, F, De Pellegrin, M, Silvani, P, Camesasca, C, Pontesilli, S, Darin, S, Ciotti, F, Sarzana, M, Consiglieri, G, Filisetti, C, Forni, G, Passerini, L, Tomasoni, D, Cesana, D, Calabria, A, Spinozzi, G, Cicalese, M, Calbi, V, Migliavacca, M, Barzaghi, F, Ferrua, F, Gallo, V, Miglietta, S, Zonari, E, Cheruku, P, Forni, C, Facchini, M, Corti, A, Gabaldo, M, Zancan, S, Gasperini, S, Rovelli, A, Boelens, J, Jones, S, Wynn, R, Baldoli, C, Montini, E, Gregori, S, Ciceri, F, Valsecchi, M, la Marca, G, Parini, R, Naldini, L, Aiuti, A, Bernardo, M, Gentner, Bernhard, Tucci, Francesca, Galimberti, Stefania, Fumagalli, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Camesasca, Chiara, Pontesilli, Silvia, Darin, Silvia, Ciotti, Francesca, Sarzana, Marina, Consiglieri, Giulia, Filisetti, Chiara, Forni, Giulia, Passerini, Laura, Tomasoni, Daniela, Cesana, Daniela, Calabria, Andrea, Spinozzi, Giulio, Cicalese, Maria-Pia, Calbi, Valeria, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Gallo, Vera, Miglietta, Simona, Zonari, Erika, Cheruku, Patali S, Forni, Claudia, Facchini, Marcella, Corti, Ambra, Gabaldo, Michela, Zancan, Stefano, Gasperini, Serena, Rovelli, Attilio, Boelens, Jaap-Jan, Jones, Simon A, Wynn, Robert, Baldoli, Cristina, Montini, Eugenio, Gregori, Silvia, Ciceri, Fabio, Valsecchi, Maria G, la Marca, Giancarlo, Parini, Rossella, Naldini, Luigi, Aiuti, Alessandro, and Bernardo, Maria-Ester
Abstract: Background Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications. Methods We are conducting an ongoing study involving eight children with MPSIH. At enrollment, the children lacked a suitable allogeneic donor and had a Developmental Quotient or Intelligence Quotient score above 70 (i.e., none had moderate or severe cognitive impairment). The children received autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with an alpha-L-iduronidase (IDUA)-encoding lentiviral vector after myeloablative conditioning. Safety and correction of blood IDUA activity up to supraphysiologic levels were the primary end points. Clearance of lysosomal storage material as well as skeletal and neurophysiological development were assessed as secondary and exploratory end points. The planned duration of the study is 5 years. Results We now report interim results. The children's mean (+/- SD) age at the time of HSPC gene therapy was 1.9 +/- 0.5 years. At a median follow-up of 2.10 years, the procedure had a safety profile similar to that known for autologous hematopoietic stem-cell transplantation. All the patients showed prompt and sustained engraftment of gene-corrected cells and had supraphysiologic blood IDUA activity within a month, which was maintained up to the latest follow-up. Urinary glycosaminoglycan (GAG) excretion decreased steeply, reaching normal levels at 12 months in four of five patients who could be evaluated. Previously undetectable levels of IDUA activity in the cerebrospinal fluid became detectable after gene therapy and were associated with local clearance of GAGs. Patients showed stable cognitive performance, stable motor skills corresponding to continued motor development, improved or stable findings on magnetic resonance imaging of the brain and spine, reduced
Published: 2021

14. ISwap: a bioinformatics tool for index switching detection in vector integration site studies

Author: De Marino, A, Calabria, A, Benedicenti, F, Antoniotti, M, Montini, E, De Marino, A, Calabria, A, Benedicenti, F, Antoniotti, M, and Montini, E
Subjects: Bioinformatic, Gene Therapy, Insertion Site
Published: 2019

15. Understanding Factors Associated With Psychomotor Subtypes of Delirium in Older Inpatients With Dementia

Author: Morandi, A, Zambon, A, Di Santo SG, Mazzone, A, Cherubini, A, Mossello, E, Bo, M, Marengoni, A, Bellelli, G, Rispoli, V, Malara, A, Spadea, F, Di Cello, S, Ceravolo, F, Fabiano, F, Chiaradia, G, Amedeo, G, Peluso, L, Taristano, A, Settembrini, V, Capomolla, D, Citrino, A, Scriva, A, Bruno, I, Secchi, R, De Martino, E, Muccinelli, R, Lupi, G, Paonessa, P, Fabbri, A, Passuti, Mt, Castellari, S, Po, A, Gaggioli, G, Varesi, M, Moneti, P, Capurso, S, Latini, V, Ghidotti, S, Riccardelli, F, Macchi, M, Rigo, R, Pascale, C, Bosio, A, Cerrato, F, Bardelli, B, Boffelli, S, Cassinadri, A, Franzoni, S, Spazzini, E, Andretto, D, Tonini, G, Andreani, L, Coralli, M, Balotta, A, Cancelliere, R, Ballardini, G, Simoncelli, M, Mancini, A, Strazzacapa, M, Cavallino, P, Fabio, S, De Filippi, F, Giudice, C, Floris, P, D’Elia, K, Dentizzi, C, Azzini, M, Cazzadori, M, Mastroeni, V, Bertassello, P, Santana, H, Benati, C, Nesta, E, Tobaldini, C, Antonioli, A, Guerini, F, Tambare, E, Mombelloni, P, Fontanini, F, Lassa, G, Pizzorni, C, Oliverio, M, Del Grosso LL, Giavedoni, C, Bidoli, G, Mazzei, B, Corsonello, A, Fusco, S, Vena, S, De Vuono, T, Maiuri, G, Fimognari, Fl, Arone, A, Sgrò, G, Nicolazzo, R, Castegnaro, E, De Rosa, S, Bazzano Sechi, R, Benvenuti, E, Del Lungo, I, Giardini, S, Giulietti, C, Di Bari, M, Barghini, E, Paoli, M, Fiordelli, I, Barucci, R, Sgrilli, F, Bertoletti, E, D'Amico, F, Caronzolo, F, Grippa, A, Lombardo, G, Pipicella, T, Satta, A, Corica, F, Prestipino, Gv, Larosa, D, Cucinotta, D, Basile, G, March, A, Nitti, Mt, Felici, A, Pavan, S, Piazzani, F, Lunelli, A, Dimori, S, Margotta, A, Soglia, T, Postacchini, D, Brunelli, R, Santini, S, Francavilla, M, Macchiati, I, Sorvillo, F, Giuli, C, Mecocci, P, Longo, A, Conestabile della Staffa, M, Perticone, F, Addesi, D, Cella Rosa, P, Bencardino, G, Falbo, T, Grillo, N, Marco, F, Mirella, F, Fantò, F, Isaia, G, Pezzilli, S, Bergamo, D, Furno, E, Rrodhe, S, Lucarini, S, Dijk, B, Dall'Acqua, F, Cappelletto, F, 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A., Morandi A., Zambon A., Di Santo S.G., Mazzone A., Cherubini A., Mossello E., Bo M., Marengoni A., Piccoli T., Bellelli G., Rispoli V., Malara A., Spadea F., Di Cello S., Ceravolo F., Fabiano F., Chiaradia G., Gabriele A., Lenino P., Andrea T., Settembrini V., Capomolla D., Citrino A., Scriva A., Bruno I., Secchi R., De Martino E., Muccinelli R., Lupi G., Paonessa P., Fabbri A., Passuti M.T., Castellari S., Po A., Gaggioli G., Varesi M., Moneti P., Capurso S., Latini V., Ghidotti S., Riccardelli F., Macchi M., Rigo R., Claudio P., Angelo B., Flavio C., Benedetta B., Boffelli S., Cassinadri A., Franzoni S., Spazzini E., Andretto D., Tonini G., Andreani L., Coralli M., Balotta A., Cancelliere R., Ballardini G., Simoncelli M., Mancini A., Strazzacapa M., Fabio S., De Filippi F., Giudice C., Dentizzi C., Azzini M., Cazzadori M., Mastroeni V., Bertassello P., Claudia Benati H.S., Nesta E., Tobaldini C., Guerini F., Elena T., Mombelloni P., Fontanini F., Gabriella L., Pizzorni C., Oliverio M., Del Grosso L.L., Giavedoni C., Bidoli G., Mazzei B., Corsonello A., Fusco S., Vena S., De Vuono T., Maiuri G., Luca F.F., Andrea A., Giovanni S., Rossella N., Castegnaro E., De Rosa S., Sechi R.B., Benvenuti E., Del Lungo I., Giardini S., Giulietti C., Mauro D.B., Eleonora B., Martina P., Irene F., Riccardo B., Federica S., Ilaria D.L., Bertoletti E., D'Amico F., Caronzolo F., Grippa A., Lombardo G., Pipicella T., Antonino S., Francesco C., Valeria P.G., Daniela L., Domenico C., Giorgio B., March A., Nitti M.T., Felici A., Pavan S., Piazzani F., Lunelli A., Dimori S., Margotta A., Soglia T., Postacchini D., Brunelli R., Santini S., Francavilla M., Macchiati I., Sorvillo F., Giuli C., Mecocci P., Longo A., Perticone F., Addesi D., Rosa P.C., Bencardino G., Falbo T., Grillo N., Marco F., Mirella F., Fanto F., Isaia G., Pezzilli S., Bergamo D., Furno E., Rrodhe S., Lucarini S., Dijk B., Dall'Acqua F., Cappelletto F., Calvani D., Becheri D., Giuseppe M., Costanza M., Vito A., Francesca B., Magherini L., Novella M., Franca B., Lucia Gambardella P.M., Valente C., Ilaria B., Alice F., Porrino P., Ceci G., Giuliana B., Michela T., Eleonora C., Ettore E., Camellini C., Servello A., Grassi A., Rozzini R., Tironi S., Grassi M.G., Troisi E., Carlo C., Simona Gabriella D.S., Flaminia F., Federica R., Beatrice P., Sofia T., Gabutto A., Quazzo L., Rosatello A., Suraci D., Tagliabue B., Perrone C., Ferrara L., Castagna A., Tremolada M.L., Giuseppe C., Stefano B., Davide O., Piano S., Serviddio G., Lo Buglio A., Gurrera T., Merlo V., Rovai C., Cotroneo A.M., Carlucci R., Abbaldo A., Monzani F., Qasem A.A., Bini G., Tafuto S., Galli G., Bruni A.C., Mancuso G., Calipari D., Giuseppe Massimiliano D.L., Bernardini B., Corsini C., Michele C., Sara D.F., Cagnin A., Fragiacomo F., Pompanin S., Piero A., Marco C., Zurlo A., Guerra G., Pala M., Menozzi L., Gatti C.D., Magon S., Roberto M., Alfredo D.G., Fabio F., Ruana T., Elisa M., Christian M., Marco P., Massimo G., Di Francesco V., Faccioli S., Pellizzari L., Giorgia F., Barbagallo G., Lunardelli M.L., Martini E., Ferrari E., Macchiarulo M., Corneli M., Bacci M., Battaglia G., Anastasio L., Lo Storto M.S., Seresin C., Simonato M., Loreggian M., Cestonaro F., Durando M., Latella R., Mazzoleni M., Russo G., Ponte M., Valchera A., Salustri G., Petritola D., Costa A., Sinforiani E., Cotta M.R., Pizio R.N., Cester A., Formilan M., Pietro B., Carbone P., Cazzaniga I., Appollonio I., Cereda D., Stabile A., Xhani R., Acampora R., Tremolizzo L., Federico P., Antonio C., Valerio P., Cesare B., Zhirajr M., Giovanni V., Maria A., Mariaelena S., Bottacchi E., Bucciantini E., Di Giovanni M., Franchi F., Lucchetti L., Mariani C., Grande G., Rapazzini P., Marco M., Romanelli G., Franco N., Alessio M., Nicola L., Laura P., Nazario P., Chiara G., Soccorso P., Andrea S., Luca B., Francesca S., Roberto A., Anna C., Fugazza L., Guerrini C., De Paduanis G., Iallonardo L., Palumbo P., Zuliani G., Ortolani B., Capatti E., Soavi C., Bianchi L., Francesconi D., Miselli A., Gloria B., Tommaso R., Chiara P., Agata M.M., Marco D.A., Luca M., Gianluca G., Suardi T., Zaccarini C., Manuela R., Mirra G., Muti E., Bottura R., Gianpaolo M., Secreto P., Bisio E., Cecchettani M., Naldi T., Pallavicino A., Pugliese M., Iozzo R.C., Grassi G., Michele B., Raffaella D.O., Fosca Q.T., Giorgio G.C., Giovanni P., Ernesto C., Mannironi A., Giorli E., Oberti S., Fierro B., Giacalone F., Mandas A., Serchisu L., Costaggiu D., Pinna E., Orru F., Mannai M., Cordioli Z., Pelizzari L., Turcato E., Arduini P., Cacace C., Chiloiro R., Cimino R., Ruberto C., Giovanni R., Pietro G., Laura G., Alberto C., Carmen R., Santo P.D., Andriolli A., Burattin G., Rossi L., Andreolli Antonino C.G., Tezza F., Maddalena P., Laura S., Crippa P., Aloisio P., Di Monda T., Malighetti A., Galbassini G., Salutis D., Ivaldi C., Russo A.M., Bennati E., Pino E., Zavarise G., Pesci A., Suigo G., Faverio P., Andrea G., Sabrina P., Zanasi M., Moniello G., Rostagno C., Cartei A., Polidori G., Ungar A., Melis M.R., Martellini E., Enrico M., Monica T., Antonella G., Giovanna L., Migliorini M., Caramelli F., Battiston B., Berardino M., Cavallo S., Alessandro M., Anna S., Lombardi B., D'Ippolito P., Furini A., Villani D., Clara R., Guarneri M., Paolucci S., Bassi A., Coiro P., De Angelis D., Morone G., Venturiero V., Palleschi L., Raganato P., Di Niro G., Rosa C.A., Loredana B., Imoscopi A., Tibaldi V., Bottignole G G., Calvi E., Clementi C., Zanocchi M., Agosta L., Nortarelli A., Provenzano G., Mari D., Romano F.Y., Rosini F., Mansi M., Rossi S., Geriatria A.R., Inzaghi L., Bonini G., Rossi P., Potena A., Lichii M., Candiani T., Grimaldi W., Bertani E., Alessandra P., Calogero P., Pinto D., Bernardi R., Nicolino F., Galetti C., Gianstefani A., Giulia C., Lorenzo M., Odetti P., Monacelli F., Prefumo M., Fiammetta M., Canepa M., Minaglia C., Paolisso G., Rizzo M.R., Prestano R., Dalise A.M., Barra D., Bosco L.D., Asprinio V., Dallape L., Perina E., Incalzi R.A., Bartoli I.R., Pluderi A., Maina A., Pecoraro E., Sciarra M., Prudente A., Paola M., Francesca M., Manuel V., Luisella C., Maria P.L., Tina S., Benini L., Levato F., Mhiuta V., Alius F., Davidoaia D., Giardini V., Garancini M., Bellamoli C., Terranova L., Bozzini C., Tosoni P., Provoli E., Cascone L., Dioli A., Ferrarin G., Bucci A., Bua G., Fenu S., Bianchi G., Casella S., Romano V., Maurizio P., Mascherona I., Belotti G., Cavaliere S., Cuni E., Merciuc N., Oberti R., Veneziani S., Capoferri E., De Bernardi E., Colombo K., Bravi M., Nicoletta N., D'Arcangelo P., Montenegro N., Montanari R., Lamanna P., Gasperini B., Isabella M., Stefania D., Gaia A., Filippo C., Palama C., Di Emidio C., Scarpini E., Arighi A., Fumagalli G., Basilico P., De Amicis Margherita M., Marta M., Diletta M., Granata A., Ranalli C., Cammilli A., Cavallini M.C., Tricca M., Natella D., Gabbani L., Tesi F., Martella L., Imbrici R., Guerrini G., Scotuzzi A.M., Sozzi F., Valenti L., Chiarello A., Monia M., Pilotto A., Prete C., Senesi B., Meta A.C., Pendenza E., Pasqualetti G., Polini A., Tognini S., Ballino E., Dell'Aquila G., Gasparrini P.M., Marotti E., Migale M., Scrimieri A., Falsetti L., Salvi A., Toigo G., Ceschia G., Rosso A., Tongiorgi C., Scarpa C., De Dominicis L., Pucci E., Renzi S., Cartechini E., Tomassini P.F., Del Gobbo M., Ugenti F., Romeo P., Nardelli A., Lauretani F., Visioli S., Montanari I., Ermini F., Giordano A., Pigato G., Simeone E., Barbujani M., Giampieri M., Amoruso R., Piccinini M., Ferrari C., Gambetti C., Sfrappini M., Semeraro L., Striuli R., Pelliccioni G., Marinelli D., Fabi K., Rossi T., Pesallaccia M., Sabbatini D., Gobbi B., Cerqua R., Tagliani G., Schlauser E., Caser L., Caramello E., Sandigliano F., Rosso G., Ferrari A., Bendini C., Luisa D.M., Casella M., Prampolini R., Scevola M., Vitale E., Roberto B., Carlo F., Sergio F., Alberto S., Daniela Z., Giulia B., Serena G., Maugeri D., Sorace R., Anzaldi M., De Gesu R., Morrone G., Davolio F., Fabbo A., Palmieri M., Zoli M., Forti P., Pirazzoli L., Fabbri E., Terenzi L., Bergolari F., Wenter C., Ruffini I., Insam M., Abraham E., Kirchlechner C., Cucinotta D., Antonino L., Basile G., Grazia A.M., Parise P., Boccali A., Amici S., Gambacorta M., Lasagni A., Lovati R., Giovinazzo F., Kimak E., Zappa P., Medici F., Lo Castro M., Mauro F., De Luca A., Sancesario G., Martorana A., Scaricamazza B., Toniolo S., Di Lorenzo F., Liguori C., Lasco A., Vita N., Giomi M., Forte F., Padovani A., Rozzini L., Ceraso A., Salvatore C., Cottino M., Vitali S., Marelli E., Tripi G., Miceli S., Urso G., Grioni G., Vezzadini G., Misaggi G., Forlani C., Avanzi S., Serena S., Claudia C., Marilena V., Alberto L., Diego G., Alessandro G., Iemolo F., Sanzaro E., D'Asta G., Proietto M., Carnemolla A., Razza G., Spadaro D., Bertolotti M., Mussi C., Neviani F., Roberto C., Valentina G., Linda M., Francesca V., Tarozzi A., Balestri F., Mannarino G., Bigolari M., Natale A., Grassi S., Bottaro C., Stefanelli S., Bovone U., Tortorolo U., Quadri R., Leone G., Ponzetto M., Frasson P., Annoni G., Bruni A., Confalonieri R., Corsi M., Moretti D., Teruzzi F., Umidi S., Mazzola P., Perego S., Persico I., Olivieri G., Bonfanti A., Hajnalka S., Galeazzi M., Massariello F., Anzuini A., Caffarra P., Barocco F., Spallazzi M., Paolo C.G., Simonetta M., Chioatto P., Bortolamei S., Soattin L., Ruotolo G., Beneamino B., Giuseppe B., Bertazzoli M., Rota E., Adobati A., Scarpa A., Granziera S., Zuccher P., Fabbro A.D., Zara D., Lo Nigro A., Franchetti L., Toniolo M., Marcuzzo C., Rollone M., Guerriero F., Sgarlata C., Masse A., Zatti G., Piatti M., Graci J., Benati G., Boschi F., Biondi M., Fiumi N., Erika T., Locatelli S.M., Mauri S., Beretta M., Margheritis L., Desideri G., Liberatore E., Carucci A.C., Bonino P., Caput M., Antonietti M.P., Polistena G., De la Pierre F., Mari M., Massignani P., Tombesi F., Selvaggio F., Verbo B., Bodoni P., Marchionni N., Sabatini T., Mussio E., Magni E., Bianchetti A., Crucitti A., Titoldini G., Cossu B., Fascendini S., Licini C., Tomasoni A., Calderazzo M., Daniela T., Valentina L., Melotti R.M., Lilli A., Buda S., Adversi M., Noro G., Turco R., Ubezio M.C., Mantovani A.R., Viola M.C., Serrati C., Pretta S., Infante M., Gentile S., D'Ambrosio V., Mazzanti P., Brambilla C., Sportelli S., Platto C., Faraci B., Quattrocchi D., Pernigotti L.M., Pisu C., Sicuro F., Zagnoni P., Ghiglia S., Mosca M., Corazzin I., Deola M., Biagini C.A., Bencini F., Cantini C., Tonon E., Pierinelli S., Onofrj M., Thomas A., Filomena B., Bonanni L., Gabriella C., Comi G., Magnani G., Santangelo R., Mazzeo S., Francesca C., Giordano C., Roberto S.A., Barbieri C., Giroldi L., Bandini F., Masina M., Malservisi S., Cicognani A., Ricca L., Piccininni M., Tassinari T., Brogi D., Sugo A., Alessandra F., Sonia M., Valerio V., Andrea U.C., Enrico C., Vera R.F., Assunta S., Gianmaria Z., Mauro P., Barone A., Razzano M., Giuseppe I., Angela B., Francesco S., Valeria D.A., Federico G., Lucia P., Antonella V., Elisabetta D.C., Cristina R., Nadia C., Maria S., Luciano A., Chiara C., Bini P., Pignata M., Enrico B., Maria V., Giovanni C., Giorgio C., Piera R., Alberto Z., Ceccon A., Magrin L., Marin S., Barbara S., Matteo M., Caterina P.M., Carla R., Federica G., Clara T., Melania C., Giampaolo B., Stefano G., Valeria G., Lucia M., Giovambattista D., Ester L., Cecilia C.A., Maurizio T., Nadia B., Grillo A., Arenare F., Tonino M., David K., Giorgio V.P., Ubaldo B., Vincenzo S., Stefano M., Marino F., Busonera Flavio M.T., Paolo A., Monica M., Francesco B., Roberto F., Paolo B., DuranteMangoni E., Testoni M., Fabio D.S., Loredana S., Valeria S., Fabiano M., Annabella D.G., Salvatore D.C., Greco A., Grazia D.O., Daniele S., Gianluca R., Renzo G., Sergio M., Morena B., Vitali M., Marina P., Paolo D.C., Cristina S., Orlandini F., La Regina M., Desiree A., Mario B., Paola P., Padulo F., Cristina M., Dario R., Giancarla M., Guido R., Elena M., Marileda N., Igor B., Nicole B., Elena R., Paolillo C., Riccardi A., Claudia B., Barbara R., Silvia V., Oliver B., Mauro C., Eleonora M., Giuseppe P., Rosaria T., Maria C., Davide D.A., Stefania C., Massimo P., Luca S., Martina D.F., Paola V., Lia S., Sandro C., Valentina D.S., Erminia B., Paola C., Romina R., Minisola S., Luciano C., Pasquale A., Ilaria L., Guglielmo S., Marco E., Sara R., Paola A., Claudio A., Francesco R., Alessandro C., Simona M., Lara F., Paola R., Simonetta C., Antonella C., Generoso U., Fernando G., Giuliano C., Emanuela S., Mariolina S., Alessandro D., Giulia L., Famularo S., Sandini M., Pinotti E., Gianotti L., Antonella B., Giulia P., Sante G., Rossi A., Rubele S., Sant S., Marco V., Danila C., Fabio R., Bandirali M.P., Nicoletta C., Laura B., Paolo T., Luciano T., Leonello A., Margherita S., Stefania D.N., Pierluigi D.S., Laura R., Fabiana T., Giovanna C., Antonino A., Felice C., Danilo F., Giovanna D.B., Francesco L., Salini S., Angela B.M., Giorgetta C., Giovanni G., Gerardo B., Silvio R., Letizia S., Davide B., Rosaria R.M., Maria D.A., Raffaele P., Palmieri V.O., Palasciano G., Belfiore A., Portincasa P., Carlo S., Alessia D., Valiani V., Carolina B., Tiziana C., Paola T., Ugo P., Giacomo P., Castellano M., Anna G., Elisa C., Federica C., Antonietta C.M., Luigi M., Fabio L., Salvatore B., Gelosa G., Viviana A.T., Piras V., Andrea C., Alessandra B., Coen D., Magliola R., Milanesio D., Muzzulini C.L., Paolo F., Marinella T., Sofia C.M., Marta B., Siano P., Capo G., Napoletano R., Cecilia P., Mancini C., Del Buono C., De Bartolomeo G., Addolorata M., Carmen C., Giovanni V.A., Moschettini G., Franco M., Daniela R., D'Amico G., Mirella P., Endrizzi C., Trotta L., Ciarambino T., Orazio Z., Emanuela T., Marta S., Thomas F., Giacomo T., Ignazio D.F., Andrea B., Giuseppe O., Emanuela F., Serena A., Elena D.I., Serena B., Erika N., Roberto S., Elena S., Manuela P., Francesca A., Angelo T., Morandi, A, Zambon, A, Di Santo, S, Mazzone, A, Cherubini, A, Mossello, E, Bo, M, Marengoni, A, Bellelli, G, Rispoli, V, Malara, A, Spadea, F, Di Cello, S, Ceravolo, F, Fabiano, F, Chiaradia, G, Gabriele, A, Lenino, P, Andrea, T, Settembrini, V, Capomolla, D, Citrino, A, Scriva, A, Bruno, I, Secchi, R, De Martino, E, Muccinelli, R, Lupi, G, Paonessa, P, Fabbri, A, Passuti, M, Castellari, S, Po, A, Gaggioli, G, Varesi, M, Moneti, P, Capurso, S, Latini, V, Ghidotti, S, Riccardelli, F, Macchi, M, Rigo, R, Claudio, P, Angelo, B, Flavio, C, Benedetta, B, Boffelli, S, Cassinadri, A, Franzoni, S, Spazzini, E, Andretto, D, Tonini, G, Andreani, L, Coralli, M, Balotta, A, Cancelliere, R, Ballardini, G, Simoncelli, M, Mancini, A, Strazzacapa, M, Fabio, S, De Filippi, F, Giudice, C, Dentizzi, C, Azzini, M, Cazzadori, M, Mastroeni, V, Bertassello, P, Claudia Benati, H, Nesta, E, Tobaldini, C, Guerini, F, Elena, T, Mombelloni, P, Fontanini, F, Gabriella, L, Pizzorni, C, Oliverio, M, Del Grosso, L, Giavedoni, C, Bidoli, G, Mazzei, B, Corsonello, A, Fusco, S, 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Migale, M, Scrimieri, A, Falsetti, L, Salvi, A, Toigo, G, Ceschia, G, Rosso, A, Tongiorgi, C, Scarpa, C, De Dominicis, L, Pucci, E, Renzi, S, Cartechini, E, Tomassini, P, Del Gobbo, M, Ugenti, F, Romeo, P, Nardelli, A, Lauretani, F, Visioli, S, Montanari, I, Ermini, F, Giordano, A, Pigato, G, Simeone, E, Barbujani, M, Giampieri, M, Amoruso, R, Piccinini, M, Ferrari, C, Gambetti, C, Sfrappini, M, Semeraro, L, Striuli, R, Pelliccioni, G, Marinelli, D, Fabi, K, Rossi, T, Pesallaccia, M, Sabbatini, D, Gobbi, B, Cerqua, R, Tagliani, G, Schlauser, E, Caser, L, Caramello, E, Sandigliano, F, Rosso, G, Ferrari, A, Bendini, C, Luisa, D, Casella, M, Prampolini, R, Scevola, M, Vitale, E, Roberto, B, Carlo, F, Sergio, F, Alberto, S, Daniela, Z, Giulia, B, Serena, G, Michele, B, Maugeri, D, Sorace, R, Anzaldi, M, De Gesu, R, Morrone, G, Davolio, F, Fabbo, A, Palmieri, M, Zoli, M, Forti, P, Pirazzoli, L, Fabbri, E, Terenzi, L, Bergolari, F, Wenter, C, Ruffini, I, Insam, M, Abraham, E, Kirchlechner, C, Cucinotta, D, Antonino, L, Basile, G, Grazia, A, Parise, P, Boccali, A, Amici, S, Gambacorta, M, Lasagni, A, Lovati, R, Giovinazzo, F, Kimak, E, Zappa, P, Medici, F, Lo Castro, M, Mauro, F, De Luca, A, Sancesario, G, Martorana, A, Scaricamazza, B, Toniolo, S, Di Lorenzo, F, Liguori, C, Lasco, A, Vita, N, Giomi, M, Forte, F, Padovani, A, Rozzini, L, Ceraso, A, Salvatore, C, Cottino, M, Vitali, S, Marelli, E, Tripi, G, Miceli, S, Urso, G, Grioni, G, Vezzadini, G, Misaggi, G, Forlani, C, Avanzi, S, Serena, S, Claudia, C, Marilena, V, Alberto, L, Diego, G, Alessandro, G, Iemolo, F, Sanzaro, E, D'Asta, G, Proietto, M, Carnemolla, A, Razza, G, Spadaro, D, Bertolotti, M, Mussi, C, Neviani, F, Roberto, C, Valentina, G, Linda, M, Francesca, V, Tarozzi, A, Balestri, F, Mannarino, G, Bigolari, M, Natale, A, Grassi, S, Bottaro, C, Stefanelli, S, Bovone, U, Tortorolo, U, Quadri, R, Leone, G, Ponzetto, M, Frasson, P, Annoni, G, Confalonieri, R, Corsi, M, Moretti, D, Teruzzi, F, Umidi, S, Mazzola, P, Perego, S, Persico, I, Olivieri, G, Bonfanti, A, Hajnalka, S, Galeazzi, M, Massariello, F, Anzuini, A, Caffarra, P, Barocco, F, Spallazzi, M, Paolo, C, Simonetta, M, Chioatto, P, Bortolamei, S, Soattin, L, Ruotolo, G, Beneamino, B, Giuseppe, B, Bertazzoli, M, Rota, E, Adobati, A, Scarpa, A, Granziera, S, Zuccher, P, Fabbro, A, Zara, D, Lo Nigro, A, Franchetti, L, Toniolo, M, Marcuzzo, C, Rollone, M, Guerriero, F, Sgarlata, C, Masse, A, Zatti, G, Piatti, M, Graci, J, Benati, G, Boschi, F, Biondi, M, Fiumi, N, Erika, T, Locatelli, S, Mauri, S, Beretta, M, Margheritis, L, Desideri, G, Liberatore, E, Carucci, A, Bonino, P, Caput, M, Antonietti, M, Polistena, G, De la Pierre, F, Mari, M, Massignani, P, Tombesi, F, Selvaggio, F, Verbo, B, Bodoni, P, Marchionni, N, Sabatini, T, Mussio, E, Magni, E, Bianchetti, A, Crucitti, A, Titoldini, G, Cossu, B, Fascendini, S, Licini, C, Tomasoni, A, Calderazzo, M, Daniela, T, Valentina, L, Melotti, R, Lilli, A, Buda, S, Adversi, M, Noro, G, Turco, R, Ubezio, M, Mantovani, A, Viola, M, Serrati, C, Pretta, S, Infante, M, Gentile, S, D'Ambrosio, V, Mazzanti, P, Brambilla, C, Sportelli, S, Platto, C, Faraci, B, Quattrocchi, D, Pernigotti, L, Pisu, C, Sicuro, F, Zagnoni, P, Ghiglia, S, Mosca, M, Corazzin, I, Deola, M, Biagini, C, Bencini, F, Cantini, C, Tonon, E, Pierinelli, S, Onofrj, M, Thomas, A, Filomena, B, Bonanni, L, Gabriella, C, Comi, G, Magnani, G, Santangelo, R, Mazzeo, S, Francesca, C, Giordano, C, Roberto, S, Barbieri, C, Giroldi, L, Bandini, F, Masina, M, Malservisi, S, Cicognani, A, Ricca, L, Piccininni, M, Tassinari, T, Brogi, D, Sugo, A, Alessandra, F, Sonia, M, Valerio, V, Andrea, U, Enrico, C, Vera, R, Assunta, S, Gianmaria, Z, Mauro, P, Barone, A, Razzano, M, Giuseppe, I, Angela, B, Francesco, S, Valeria, D, Federico, G, Lucia, P, Antonella, V, Elisabetta, D, Cristina, R, Nadia, C, Maria, S, Luciano, A, Chiara, C, Bini, P, Pignata, M, Enrico, B, Maria, V, Giovanni, C, Giorgio, C, Piera, R, Alberto, Z, Ceccon, A, Magrin, L, Marin, S, Barbara, S, Matteo, M, Caterina, P, Carla, R, Federica, G, Clara, T, Melania, C, Giampaolo, B, Stefano, G, Valeria, G, Lucia, M, Giovambattista, D, Ester, L, Cecilia, C, Maurizio, T, Nadia, B, Grillo, A, Arenare, F, Tonino, M, David, K, Giorgio, V, Ubaldo, B, Vincenzo, S, Stefano, M, Marino, F, Busonera Flavio, M, Paolo, A, Monica, M, Francesco, B, Roberto, F, Paolo, B, Durantemangoni, E, Testoni, M, Fabio, D, Loredana, S, Valeria, S, Fabiano, M, Annabella, D, Salvatore, D, Greco, A, Grazia, D, Daniele, S, Gianluca, R, Renzo, G, Sergio, M, Morena, B, Vitali, M, Marina, P, Paolo, D, Cristina, S, Orlandini, F, La Regina, M, Desiree, A, Mario, B, Paola, P, Padulo, F, Cristina, M, Dario, R, Giancarla, M, Guido, R, Elena, M, Marileda, N, Igor, B, Nicole, B, Elena, R, Paolillo, C, Riccardi, A, Claudia, B, Barbara, R, Silvia, V, Oliver, B, Mauro, C, Eleonora, M, Giuseppe, P, Rosaria, T, Maria, C, Davide, D, Stefania, C, Massimo, P, Luca, S, Martina, D, Paola, V, Lia, S, Sandro, C, Valentina, D, Erminia, B, Paola, C, Romina, R, Minisola, S, Luciano, C, Pasquale, A, Ilaria, L, Guglielmo, S, Marco, E, Sara, R, Paola, A, Claudio, A, Francesco, R, Alessandro, C, Simona, M, Lara, F, Paola, R, Simonetta, C, Antonella, C, Generoso, U, Fernando, G, Giuliano, C, Emanuela, S, Mariolina, S, Alessandro, D, Giulia, L, Famularo, S, Sandini, M, Pinotti, E, Gianotti, L, Antonella, B, Giulia, P, Sante, G, Rossi, A, Rubele, S, Sant, S, Marco, V, Danila, C, Fabio, R, Bandirali, M, Nicoletta, C, Laura, B, Paolo, T, Luciano, T, Leonello, A, Margherita, S, Pierluigi, D, Laura, R, Fabiana, T, Giovanna, C, Antonino, A, Felice, C, Danilo, F, Giovanna, D, Francesco, L, Salini, S, Giorgetta, C, Giovanni, G, Gerardo, B, Silvio, R, Letizia, S, Davide, B, Rosaria, R, Maria, D, Raffaele, P, Palmieri, V, Palasciano, G, Belfiore, A, Portincasa, P, Carlo, S, Alessia, D, Valiani, V, Carolina, B, Tiziana, C, Paola, T, Ugo, P, Giacomo, P, Castellano, M, Anna, G, Elisa, C, Federica, C, Antonietta, C, Luigi, M, Fabio, L, Salvatore, B, Gelosa, G, Viviana, A, Piras, V, Andrea, C, Alessandra, B, Coen, D, Magliola, R, Milanesio, D, Muzzulini, C, Paolo, F, Marinella, T, Sofia, C, Marta, B, Siano, P, Capo, G, Napoletano, R, Cecilia, P, Mancini, C, Del Buono, C, De Bartolomeo, G, Addolorata, M, Carmen, C, Moschettini, G, Franco, M, Daniela, R, D'Amico, G, Mirella, P, Endrizzi, C, Trotta, L, Ciarambino, T, Orazio, Z, Emanuela, T, Marta, S, Thomas, F, Giacomo, T, Ignazio, D, Andrea, B, Giuseppe, O, Emanuela, F, Serena, A, Elena, D, Serena, B, Erika, N, Elena, S, Manuela, P, Francesca, A, Angelo, T, Di Santo SG, Rizzo, Maria Rosaria, Paolisso, Giuseppe, and Italian Study Group on Delirium, (ISGoD).
Subjects: Rehabilitation hospital, medicine.medical_specialty, Urinary system, Socio-culturale, dementia, elderly, Motor subtypes of delirium, Aged, Cross-Sectional Studies, Humans, Inpatients, Italy, Delirium, Dementia, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, mental disorders, medicine, Motor subtypes of delirium, dementia, elderly, dementia, elderly, Motor subtypes of delirium, 030212 general & internal medicine, LS4_4, Medical prescription, General Nursing, Psychomotor learning, business.industry, Health Policy, Medical record, Memantine, General Medicine, medicine.disease, Settore MED/26 - NEUROLOGIA, Geriatrics and Gerontology, medicine.symptom, business, 030217 neurology & neurosurgery, medicine.drug
Abstract: Objectives Few studies have analyzed factors associated with delirium subtypes. In this study, we investigate factors associated with subtypes of delirium only in patients with dementia to provide insights on the possible prevention and treatments. Design This is a cross-sectional study nested in the "Delirium Day" study, a nationwide Italian point-prevalence study. Setting and participants Older patients admitted to 205 acute and 92 rehabilitation hospital wards. Measures Delirium was evaluated with the 4-AT and the motor subtypes with the Delirium Motor Subtype Scale. Dementia was defined by the presence of a documented diagnosis in the medical records and/or prescription of acetylcholinesterase inhibitors or memantine prior to admission. Results Of the 1057 patients with dementia, 35% had delirium, with 25.6% hyperactive, 33.1% hypoactive, 34.5% mixed, and 6.7% nonmotor subtype. There were higher odds of having venous catheters in the hypoactive (OR 1.82, 95% CI 1.18-2.81) and mixed type of delirium (OR 2.23, CI 1.43-3.46), whereas higher odds of urinary catheters in the hypoactive (OR 2.91, CI 1.92-4.39), hyperactive (OR 1.99, CI 1.23-3.21), and mixed types of delirium (OR 2.05, CI 1.36-3.07). We found higher odds of antipsychotics both in the hyperactive (OR 2.87, CI 1.81-4.54) and mixed subtype (OR 1.84, CI 1.24-2.75), whereas higher odds of antibiotics was present only in the mixed subtype (OR 1.91, CI 1.26-2.87). Conclusions and implications In patients with dementia, the mixed delirium subtype is the most prevalent followed by the hypoactive, hyperactive, and nonmotor subtype. Motor subtypes of delirium may be triggered by clinical factors, including the use of venous and urinary catheters, and the use of antipsychotics. Future studies are necessary to provide further insights on the possible pathophysiology of delirium in patients with dementia and to address the optimization of the management of potential risk factors.
Published: 2020

16. Sleeping Beauty-engineered CAR T cells achieve anti-leukemic activity without severe toxicities

Author: Magnani, C, Gaipa, G, Lussana, F, Belotti, D, Gritti, G, Napolitano, S, Matera, G, Cabiati, B, Buracchi, C, Borleri, G, Fazio, G, Zaninelli, S, Tettamanti, S, Cesana, S, Colombo, V, Quaroni, M, Cazzaniga, G, Rovelli, A, Biagi, E, Galimberti, S, Calabria, A, Benedicenti, F, Montini, E, Ferrari, S, Introna, M, Balduzzi, A, Valsecchi, M, Dastoli, G, Rambaldi, A, Biondi, A, Magnani, Chiara F, Gaipa, Giuseppe, Lussana, Federico, Belotti, Daniela, Gritti, Giuseppe, Napolitano, Sara, Matera, Giada, Cabiati, Benedetta, Buracchi, Chiara, Borleri, Gianmaria, Fazio, Grazia, Zaninelli, Silvia, Tettamanti, Sarah, Cesana, Stefania, Colombo, Valentina, Quaroni, Michele, Cazzaniga, Giovanni, Rovelli, Attilio, Biagi, Ettore, Galimberti, Stefania, Calabria, Andrea, Benedicenti, Fabrizio, Montini, Eugenio, Ferrari, Silvia, Introna, Martino, Balduzzi, Adriana, Valsecchi, Maria Grazia, Dastoli, Giuseppe, Rambaldi, Alessandro, Biondi, Andrea, Magnani, C, Gaipa, G, Lussana, F, Belotti, D, Gritti, G, Napolitano, S, Matera, G, Cabiati, B, Buracchi, C, Borleri, G, Fazio, G, Zaninelli, S, Tettamanti, S, Cesana, S, Colombo, V, Quaroni, M, Cazzaniga, G, Rovelli, A, Biagi, E, Galimberti, S, Calabria, A, Benedicenti, F, Montini, E, Ferrari, S, Introna, M, Balduzzi, A, Valsecchi, M, Dastoli, G, Rambaldi, A, Biondi, A, Magnani, Chiara F, Gaipa, Giuseppe, Lussana, Federico, Belotti, Daniela, Gritti, Giuseppe, Napolitano, Sara, Matera, Giada, Cabiati, Benedetta, Buracchi, Chiara, Borleri, Gianmaria, Fazio, Grazia, Zaninelli, Silvia, Tettamanti, Sarah, Cesana, Stefania, Colombo, Valentina, Quaroni, Michele, Cazzaniga, Giovanni, Rovelli, Attilio, Biagi, Ettore, Galimberti, Stefania, Calabria, Andrea, Benedicenti, Fabrizio, Montini, Eugenio, Ferrari, Silvia, Introna, Martino, Balduzzi, Adriana, Valsecchi, Maria Grazia, Dastoli, Giuseppe, Rambaldi, Alessandro, and Biondi, Andrea
Abstract: BACKGROUND. Chimeric antigen receptor (CAR) T cell immunotherapy has resulted in complete remission (CR) and durable response in highly refractory patients. However, logistical complexity and high costs of manufacturing autologous viral products limit CAR T cell availability. METHODS. We report the early results of a phase I/II trial in B cell acute lymphoblastic leukemia (B-ALL) patients relapsed after allogeneic hematopoietic stem cell transplantation (HSCT) using donor-derived CD19 CAR T cells generated with the Sleeping Beauty (SB) transposon and differentiated into cytokine-induced killer (CIK) cells. RESULTS. The cellular product was produced successfully for all patients from the donor peripheral blood (PB) and consisted mostly of CD3+ lymphocytes with 43% CAR expression. Four pediatric and 9 adult patients were infused with a single dose of CAR T cells. Toxicities reported were 2 grade I and 1 grade II cytokine-release syndrome (CRS) cases at the highest dose in the absence of graft-versus-host disease (GVHD), neurotoxicity, or dose-limiting toxicities. Six out of 7 patients receiving the highest doses achieved CR and CR with incomplete blood count recovery (CRi) at day 28. Five out of 6 patients in CR were also minimal residual disease negative (MRD–). Robust expansion was achieved in the majority of the patients. CAR T cells were measurable by transgene copy PCR up to 10 months. Integration site analysis showed a positive safety profile and highly polyclonal repertoire in vitro and at early time points after infusion. CONCLUSION. SB-engineered CAR T cells expand and persist in pediatric and adult B-ALL patients relapsed after HSCT. Antileukemic activity was achieved without severe toxicities.
Published: 2020

17. GMP production of anti-tumor cytotoxic T-cell lines for adoptive T-cell therapy in patients with solid neoplasia

Author: Turin, I., Pedrazzoli, P., Tullio, C., Montini, E., Carmela La Grotteria, M., Schiavo, R., Perotti, C., Locatelli, F., Carretto, E., Maccario, R., Siena, S., and Montagna, D.
Published: 2007
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18. Characterisation of CTL directed towards non-inherited maternal alloantigens in human cord blood

Author: Moretta, A, Locatelli, F, Mingrat, G, Rondini, G, Montagna, D, Comoli, P, Gandossini, S, Montini, E, Labirio, M, and Maccario, R
Published: 1999
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19. Ex vivo generation of donor-derived WT1-specific cytotoxic T-cells able to lyse patientʼs leukaemia blasts for adoptive immunotherapy after allogeneic haematopoietic stem cell transplantation: P1045

Author: Turin, I., Lisini, D., Montini, E., Caliogna, L., Moretta, A., Bonetti, F., Zavras, N., Grecchi, I., Zecca, M., Maccario, R., and Montagna, D.
Published: 2011

20. AB0052 ROLE OF TRAINED IMMUNITY AND IMMUNOMETABOLISM IN THE PATHOGENESIS OF ERDHEIM-CHESTER DISEASE

Author: Biavasco, R., primary, Molteni, R., additional, Stefanoni, D., additional, Ferrarini, M., additional, Ferrero, E., additional, Cenci, S., additional, Cardaci, S., additional, Boletta, A., additional, Cassina, L., additional, Di Stefano, G., additional, Dominguez Andres, J., additional, Doglioni, C., additional, Nemkov, T., additional, Merelli, I., additional, D’alessandro, A., additional, Montini, E., additional, Netea, M., additional, Dagna, L., additional, and Cavalli, G., additional
Published: 2020
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21. VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites

Author: Spinozzi, G, Calabria, A, Brasca, S, Beretta, S, Merelli, I, Milanesi, L, Montini, E, SPINOZZI, GIULIO, CALABRIA, ANDREA, Montini, E., Spinozzi, G, Calabria, A, Brasca, S, Beretta, S, Merelli, I, Milanesi, L, Montini, E, SPINOZZI, GIULIO, CALABRIA, ANDREA, and Montini, E.
Abstract: Background: Bioinformatics tools designed to identify lentiviral or retroviral vector insertion sites in the genome of host cells are used to address the safety and long-term efficacy of hematopoietic stem cell gene therapy applications and to study the clonal dynamics of hematopoietic reconstitution. The increasing number of gene therapy clinical trials combined with the increasing amount of Next Generation Sequencing data, aimed at identifying integration sites, require both highly accurate and efficient computational software able to correctly process "big data" in a reasonable computational time. Results: Here we present VISPA2 (Vector Integration Site Parallel Analysis, version 2), the latest optimized computational pipeline for integration site identification and analysis with the following features: (1) the sequence analysis for the integration site processing is fully compliant with paired-end reads and includes a sequence quality filter before and after the alignment on the target genome; (2) an heuristic algorithm to reduce false positive integration sites at nucleotide level to reduce the impact of Polymerase Chain Reaction or trimming/alignment artifacts; (3) a classification and annotation module for integration sites; (4) a user friendly web interface as researcher front-end to perform integration site analyses without computational skills; (5) the time speedup of all steps through parallelization (Hadoop free). Conclusions: We tested VISPA2 performances using simulated and real datasets of lentiviral vector integration sites, previously obtained from patients enrolled in a hematopoietic stem cell gene therapy clinical trial and compared the results with other preexisting tools for integration site analysis. On the computational side, VISPA2 showed a>6-fold speedup and improved precision and recall metrics (1 and 0.97 respectively) compared to previously developed computational pipelines. These performances indicate that VISPA2 is a fast, reliable a
Published: 2017

22. HIV-1-mediated insertional activation of STAT5B and BACH2 trigger viral reservoir in T regulatory cells

Author: Cesana, D, Santoni De Sio, F, Rudilosso, L, Gallina, P, Calabria, A, Beretta, S, Merelli, I, Bruzzesi, E, Passerini, L, Nozza, S, Vicenzi, E, Poli, G, Gregori, S, Tambussi, G, Montini, E, Montini, E., CALABRIA, ANDREA, BERETTA, STEFANO, MERELLI, IVAN, PASSERINI, LAURA, Cesana, D, Santoni De Sio, F, Rudilosso, L, Gallina, P, Calabria, A, Beretta, S, Merelli, I, Bruzzesi, E, Passerini, L, Nozza, S, Vicenzi, E, Poli, G, Gregori, S, Tambussi, G, Montini, E, Montini, E., CALABRIA, ANDREA, BERETTA, STEFANO, MERELLI, IVAN, and PASSERINI, LAURA
Abstract: HIV-1 insertions targeting BACH2 or MLK2 are enriched and persist for decades in hematopoietic cells from patients under combination antiretroviral therapy. However, it is unclear how these insertions provide such selective advantage to infected cell clones. Here, we show that in 30/87 (34%) patients under combination antiretroviral therapy, BACH2, and STAT5B are activated by insertions triggering the formation of mRNAs that contain viral sequences fused by splicing to their first protein-coding exon. These chimeric mRNAs, predicted to express full-length proteins, are enriched in T regulatory and T central memory cells, but not in other T lymphocyte subsets or monocytes. Overexpression of BACH2 or STAT5B in primary T regulatory cells increases their proliferation and survival without compromising their function. Hence, we provide evidence that HIV-1-mediated insertional activation of BACH2 and STAT5B favor the persistence of a viral reservoir in T regulatory cells in patients under combination antiretroviral therapy.
Published: 2017

23. In vitro separation of GVL effect and GVHR from anti-leukaemia CTL lines: a perspective for adoptive immunotherapy after allogeneic HSCT

Author: Montagna, D., Locatelli, F., Daudt, L., Turin, I., Montini, E., Lisini, D., Zecca, M., Cerutti, P., Moretta, A., Comoli, P., and Maccario, R.
Published: 2004

24. Phase-I-Studie zur 2-mal wöchentlichen Behandlung mit Oxaliplatin (L-OHP), Fluorouracil (5-FU) und Folinsäure (FA) bei vorbehandelten Patienten mit metastasierendem Kolorektalkarzinom (MCRC): 2231

Author: Marini, G., Ferrari, V. D., Simoncini, E., Marpicati, P., Montini, E., Rangoni, G., Mambrini, A., Pagani, M., and Vassalli, L.
Published: 2001

25. PHASE I-II STUDY OF GEMCITABINE (GEM) PLUS FLUOROURACIL (5-FU) AND FOLINIC ACID (FA) IN PATIENTS WITH ADVANCED BREAST CANCER (ABC).

Author: Simoncini, E, Ferrari, V D, Marpicati, P, Montini, E, Rangoni, G, and Marini, G
Published: 2000

26. IS THERE A CUMULATIVE DOSE LIMITING TOXICITY IN BIPHOSPHONATES THERAPY IN MANAGEMENT OF SYMPTOMATIC HYPERCALCEMIA? OUR EXPERIENCE.

Author: Marini, G, Simoncini, E, Marpicati, P, Montini, E, Rangoni, G, and Ferrari, V D
Published: 2000

27. New Graph-Based Algorithm for Comprehensive Identification and Tracking Retroviral Integration Sites

Author: Calabria, A, Beretta, S, Merelli, I, Spinozzi, G, Brasca, S, Benedicenti, F, Tenderini, E, Biffi, A, Montini, E, CALABRIA, ANDREA, BERETTA, STEFANO, MERELLI, IVAN, SPINOZZI, GIULIO, Montini, E., Calabria, A, Beretta, S, Merelli, I, Spinozzi, G, Brasca, S, Benedicenti, F, Tenderini, E, Biffi, A, Montini, E, CALABRIA, ANDREA, BERETTA, STEFANO, MERELLI, IVAN, SPINOZZI, GIULIO, and Montini, E.
Published: 2016

28. PF436 GMP MANUFACTURING OF ALLOGENEIC CD19 CHIMERIC ANTIGEN RECEPTOR (CAR) CYTOKINE INDUCED KILLER (CIK) CELLS WITH SLEEPING BEAUTY (SB) TRANSPOSON FOR ADOPTIVE IMMUNOTHERAPY

Author: Magnani, C.F., primary, Gaipa, G., additional, Belotti, D., additional, Matera, G., additional, Tettamanti, S., additional, Cabiati, B., additional, Cesana, S., additional, Colombo, V., additional, Cazzaniga, G., additional, Fazio, G., additional, Buracchi, C., additional, Rigamonti, S., additional, Rovelli, A., additional, Balduzzi, A., additional, Napolitano, S., additional, Montini, E., additional, Borleri, G.M., additional, Gritti, G., additional, Lussana, F., additional, Introna, M., additional, Rambaldi, A., additional, Dastoli, G., additional, and Biondi, A., additional
Published: 2019
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29. New Graph-Based Algorithm for Comprehensive Identification and Tracking Retroviral Integration Sites

Author: CALABRIA, ANDREA, BERETTA, STEFANO, MERELLI, IVAN, SPINOZZI, GIULIO, Brasca, S, Benedicenti, F, Tenderini, E, Biffi, A, Montini, E., Calabria, A, Beretta, S, Merelli, I, Spinozzi, G, Brasca, S, Benedicenti, F, Tenderini, E, Biffi, A, and Montini, E
Subjects: Retroviral Integration Sites, Cell Therapy
Published: 2016

30. Confirming and investigating the role of breast cancer PIK3CA-ERBB2 genes in Anti-Cancer Drug Resistance with a new framework for the inference of cancer progression graphs using vector integration sites data

Author: Spinozzi, G, Calabria, A, Caravagna, G, Graudenzi, A, Ramazzotti, A, Antoniotti, M, Mauri, G, Montini, E, Spinozzi, G, Calabria, A, Caravagna, G, Graudenzi, A, Ramazzotti, A, Antoniotti, M, Mauri, G, and Montini, E
Published: 2016

31. adLIMS: A customized open source software that allows bridging clinical and basic molecular research studies

Author: Calabria, A, Spinozzi, G, Benedicenti, F, Tenderini, E, Montini, E, CALABRIA, ANDREA, SPINOZZI, GIULIO, Montini, E., Calabria, A, Spinozzi, G, Benedicenti, F, Tenderini, E, Montini, E, CALABRIA, ANDREA, SPINOZZI, GIULIO, and Montini, E.
Abstract: Background: Many biological laboratories that deal with genomic samples are facing the problem of sample tracking, both for pure laboratory management and for efficiency. Our laboratory exploits PCR techniques and Next Generation Sequencing (NGS) methods to perform high-throughput integration site monitoring in different clinical trials and scientific projects. Because of the huge amount of samples that we process every year, which result in hundreds of millions of sequencing reads, we need to standardize data management and tracking systems, building up a scalable and flexible structure with web-based interfaces, which are usually called Laboratory Information Management System (LIMS). Methods: We started collecting end-users' requirements, composed of desired functionalities of the system and Graphical User Interfaces (GUI), and then we evaluated available tools that could address our requirements, spanning from pure LIMS to Content Management Systems (CMS) up to enterprise information systems. Our analysis identified ADempiere ERP, an open source Enterprise Resource Planning written in Java J2EE, as the best software that also natively implements some highly desirable technological advances, such as the high usability and modularity that grants high use-case flexibility and software scalability for custom solutions. Results: We extended and customized ADempiere ERP to fulfil LIMS requirements and we developed adLIMS. It has been validated by our end-users verifying functionalities and GUIs through test cases for PCRs samples and pre-sequencing data and it is currently in use in our laboratories. adLIMS implements authorization and authentication policies, allowing multiple users management and roles definition that enables specific permissions, operations and data views to each user. For example, adLIMS allows creating sample sheets from stored data using available exporting operations. This simplicity and process standardization may avoid manual errors and infor
Published: 2015

32. HIV-1 mediated insertional activation of STAT5B and BACH2 promotes the formation of a viral reservoir in T regulatory cells

Author: Cesana, D., primary, Santoni de Sio, F., additional, Rudilosso, L., additional, Gallina, P., additional, Calabria, A., additional, Bruzzesi, E., additional, Passerini, L., additional, Nozza, S., additional, Vicenzi, E., additional, Poli, G., additional, Gregori, S., additional, Tambussi, G., additional, and Montini, E., additional
Published: 2017
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33. VISPA: a computational pipeline for the identification and analysis of genomic vector integration sites

Author: Calabria, A, Leo, S, Benedicenti, F, Cesana, D, Spinozzi, G, Orsini, M, Merella, S, Stupka, E, Zanetti, G, Montini, E, CALABRIA, ANDREA, SPINOZZI, GIULIO, Montini, E., Calabria, A, Leo, S, Benedicenti, F, Cesana, D, Spinozzi, G, Orsini, M, Merella, S, Stupka, E, Zanetti, G, Montini, E, CALABRIA, ANDREA, SPINOZZI, GIULIO, and Montini, E.
Abstract: The analysis of the genomic distribution of viral vector genomic integration sites is a key step in hematopoietic stem cell-based gene therapy applications, allowing to assess both the safety and the efficacy of the treatment and to study the basic aspects of hematopoiesis and stem cell biology. Identifying vector integration sites requires ad-hoc bioinformatics tools with stringent requirements in terms of computational efficiency, flexibility, and usability. We developed VISPA (Vector Integration Site Parallel Analysis), a pipeline for automated integration site identification and annotation based on a distributed environment with a simple Galaxy web interface. VISPA was successfully used for the bioinformatics analysis of the follow-up of two lentiviral vector-based hematopoietic stem-cell gene therapy clinical trials. Our pipeline provides a reliable and efficient tool to assess the safety and efficacy of integrating vectors in clinical settings
Published: 2014

34. Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding

Author: Biasco L, Scala S., Dionisio F., Calabria A., Basso Ricci L., Scaramuzza S., Baricordi C., Giannelli S., Neduva V. X., Dow D. J., Pellin D., Di Serio C., Montini E., Naldini L., Aiuti A., VICARD, Paola, Biasco, L, Scala, S., Dionisio, F., Calabria, A., Basso Ricci, L., Scaramuzza, S., Baricordi, C., Giannelli, S., Neduva, V. X., Dow, D. J., Pellin, D., Vicard, Paola, Di Serio, C., Montini, E., Naldini, L., and Aiuti, A.
Published: 2014

35. Lentiviral Vector-based Insertional Mutagenesis Identifies Genes Involved in the Resistance to Targeted Anticancer Therapies

Author: Ranzani M, Annunziato S, Calabria A, Brasca S, Benedicenti F, Gallina P, Montini E., NALDINI , LUIGI, Ranzani, M, Annunziato, S, Calabria, A, Brasca, S, Benedicenti, F, Gallina, P, Naldini, Luigi, and Montini, E.
Published: 2014

36. OP0179 Brafv600e promotes myeloid skewing in multisystemic langerhans cell histiocytosis humanized mouse model

Author: Biavasco, R, primary, Norelli, M, additional, Camisa, B, additional, Cesana, D, additional, Gallina, P, additional, Gentner, B, additional, Ponzoni, M, additional, Dagna, L, additional, Bondanza, A, additional, and Montini, E, additional
Published: 2017
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37. Preclinical evaluation of donor-derived sleeping beauty modified CD19CAR+ lymphocytes for the treatment of acute lymphoblastic leukemia

Author: Magnani, C., primary, Mezzanotte, C., additional, Cappuzzello, C., additional, Benedicenti, F., additional, Belotti, D., additional, Cabiati, B., additional, Bardini, M., additional, Fazio, G., additional, Cazzaniga, G., additional, Cooper, L., additional, Montini, E., additional, Gaipa, G., additional, Biondi, A., additional, and Biagi, E., additional
Published: 2017
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38. The genotoxic potential of lentiviral vector integration is modulated by the interplay between vector design and mouse genotype

Author: Cesana D., Ranzani M., Volpin M., Bartholomae C., Merella S., Benedicenti F., Sergi L. Sergi, SanVito F., Brombin C, Nonis A., Di Serio C., Doglioni C., VonKalle C., Schmidt M., Naldini L., Montini E., Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Merella, S., Benedicenti, F., Sergi, Sergi L., Sanvito, F., Brombin, C, Nonis, A., Di Serio, C., Doglioni, C., Vonkalle, C., Schmidt, M., Naldini, L., and Montini, E.
Published: 2013

39. Tracking the Fate and Activity of Individual HSC and Memory Stem T Cell Clones in GT Patients through Insertional Tagging

Author: Biasco L, Baricordi C, Cieri N, Bartholomae C, Calabria A, Brigida I, Fronza R, Pellin D, Di Serio C, Montini E, von Kalle C, Schmidt M, Aiuti A., BONINI , MARIA CHIARA, Biasco, L, Baricordi, C, Cieri, N, Bartholomae, C, Calabria, A, Brigida, I, Fronza, R, Pellin, D, Di Serio, C, Montini, E, Bonini, MARIA CHIARA, von Kalle, C, Schmidt, M, and Aiuti, A.
Published: 2012

40. New liver cancer genes identified by lentiviral vector-based insertional mutagenesis in mice are associated to differential survival in hepatocellular carcinoma patients

Author: Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, Montini E, Ranzani, M, Cesana, D, Bartholomae, Cc, Sanvito, F, Pala, M, Benedicenti, F, Gallina, P, Sergi, L, Merella, S, Bulfone, A, Doglioni, C, von Kalle, C, Kim, Yj, Schmidt, M, Tonon, G, Naldini, L, and Montini, E
Published: 2012

41. Immunotherapy of acute leukemia by chimeric antigen receptormodified lymphocytes using an improved Sleeping Beauty transposon platform

Author: Magnani, C, Turazzi, N, Benedicenti, F, Calabria, A, Tenderini, E, Tettamanti, S, Giordano Attianese, G, Cooper, L, Aiuti, A, Montini, E, Biondi, A, Biagi, E, Magnani, C, Turazzi, N, Benedicenti, F, Calabria, A, Tenderini, E, Tettamanti, S, Giordano Attianese, G, Cooper, L, Aiuti, A, Montini, E, Biondi, A, and Biagi, E
Abstract: Chimeric antigen receptor (CAR)-modified T-cell adoptive immunotherapy is a remarkable therapeutic option proven effective in the treatment of hematological malignancies. In order to optimize cell manufacturing, we sought to develop a novel clinical-grade protocol to obtain CAR-modified cytokine-induced killer cells (CIKs) using the Sleeping Beauty (SB) transposon system. Administration of irradiated PBMCs overcame cell death of stimulating cells induced by non-viral transfection, enabling robust gene transfer together with efficient T-cell expansion. Upon single stimulation, we reached an average of 60% expression of CD123- and CD19- specific 3rd generation CARs (CD28/OX40/TCRzeta). Furthermore, modified cells displayed persistence of cell subsets with memory phenotype, specific and effective lytic activity against leukemic cell lines and primary blasts, cytokine secretion, and proliferation. Adoptive transfer of CD123.CAR or CD19.CAR lymphocytes led to a significant anti-tumor response against acute myelogenous leukemia (AML) and acute lymphoblastic leukemia (ALL) disseminated diseases in NSG mice. Notably, we found no evidence of integration enrichment near cancer genes and transposase expression at the end of the differentiation. Taken all together, our findings describe a novel donor-derived non-viral CAR approach that may widen the repertoire of available methods for T cell-based immunotherapy.
Published: 2016

42. In vivo Correction of a Mouse Model of Hereditary Tyrosinemia Type I by the Sleeping Beauty (SB) Transposon

Author: Montini, E., Noll, M., Morcinek, N., Major, A., Finegold, M., Kay, M., and Grompe, M.
Subjects: Tyrosine metabolism -- Genetic aspects, Genetic disorders -- Research, Biological sciences
Published: 2001

43. Identification of new human liver cancer genes by a novel lentiviral vector-based insertional mutagenesis approach in three mouse models of hepatocarcinogenesis

Author: Ranzani M, Cesana D, Bartholomae C, Sanvito F, Pala M, Benedicenti F, Sergi LS, Bulfone A, Doglioni C, von Kalle C, Schmidt M, Tonon G, Naldini L, Montini E, Ranzani, M, Cesana, D, Bartholomae, C, Sanvito, F, Pala, M, Benedicenti, F, Sergi, L, Bulfone, A, Doglioni, C, von Kalle, C, Schmidt, M, Tonon, G, Naldini, L, and Montini, E
Published: 2011

44. Lentiviral vector-based insertional mutagenesis identifies new clinically relevant cancer genes involved in the pathogenesis of hepatocellular carcinoma

Author: Ranzani M, Cesana D, Bartholomae C, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi L, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, Montini E, Ranzani, M, Cesana, D, Bartholomae, C, Sanvito, F, Pala, M, Benedicenti, F, Gallina, P, Sergi, L, Merella, S, Bulfone, A, Doglioni, C, von Kalle, C, Kim, Yj, Schmidt, M, Tonon, G, Naldini, L, and Montini, E
Published: 2011

45. Tracking hematopoietic stem cell fate in humans by retroviral tagging

Author: Biasco, L., Baricordi, C., Dionisio, F., Bartholomae, C., Brigida, I., Scaramuzza, S., Fronza, R., Merella, S., Alessandro Ambrosi, Pellin, D., Di Serio, C., Montini, E., Kalle, C., Schmidt, M., Aiuti, A., Biasco, L, Baricordi, C, Dionisio, F, Bartholomae, C, Brigida, I, Scaramuzza, S, Fronza, R, Merella, S, Ambrosi, Alessandro, Pellin, D, DI SERIO, Mariaclelia, Montini, E, Von Kalle, C, Schmidt, M, and Aiuti, A.
Published: 2011

46. In vivo models to assess the risk of insertional mutagenesis in the liver upon vector systemic delivery

Author: Ranzani M, Cesana D, Schmidt M, Sanvito F, Benedicenti F, Bartholoma C, Ponzoni M, Cantore A, Sergi LS, Doglioni C, VonKalle C, Naldini L, Montini E, Ranzani, M, Cesana, D, Schmidt, M, Sanvito, F, Benedicenti, F, Bartholoma, C, Ponzoni, M, Cantore, A, Sergi, L, Doglioni, C, Vonkalle, C, Naldini, L, and Montini, E
Published: 2009

47. Characterization of potential genomic 'safe harbor' for efficient targeted gene addition with zinc finger nucleases

Author: Lombardo A, Cesana D, Genovese P, Maruggi G, Provasi E, Mavilio F, Holmes MC, Gregory PD, Montini E, Naldini L., BONINI , MARIA CHIARA, Lombardo, A, Cesana, D, Genovese, P, Maruggi, G, Provasi, E, Bonini, MARIA CHIARA, Mavilio, F, Holmes, Mc, Gregory, Pd, Montini, E, and Naldini, L.
Published: 2009

48. Mouse Models To Assess the Risk of Vector Insertional Mutagenesis upon Systemic Delivery

Author: Ranzani M, Cesana D, Schmidt M, Sanvito F, Benedicenti F, Bartholoma C, Ponzoni M, Cantore A, Sergi LS, Doglioni C, VonKalle C, Naldini L, Montini E, Ranzani, M, Cesana, D, Schmidt, M, Sanvito, F, Benedicenti, F, Bartholoma, C, Ponzoni, M, Cantore, A, Sergi, L, Doglioni, C, Vonkalle, C, Naldini, L, and Montini, E
Published: 2009

49. Efficient lentiviral vector-mediated delivery of hARSA in the brain of juvenile non-human primates

Author: Meneghini, V., Lattanzi, A., Tiradani, L., Bravo, G., Morena, Francesco, Martino, Sabata, Calabria, A., Benedicenti, F., Bringas, J., and Montini E. Bankiewicz K., Gritti Angela
Published: 2014

50. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection

Author: Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C., Ranzani, M., Benedicenti, F., Sergi, Ls, Alessandro Ambrosi, Ponzoni, M., Doglioni, C., Di Serio, C., Kalle, C., Naldini, L., Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Bartholomae, C, Ranzani, M, Benedicenti, F, Sergi, L, Ambrosi, A, Ponzoni, M, Doglioni, C, Di Serio, C, von Kalle, C, and Naldini, L
Published: 2008

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