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1. Long-term safety of hyaluronidase-facilitated subcutaneous immunoglobulin 10%: a European post-authorization study.

Author

Ellerbroek, Pauline, Hanitsch, Leif, Witte, Torsten, Lougaris, Vassilios, Hagen, P, Paassen, Pieter, Chen, Jie, Fielhauer, Katharina, McCoy, Barbara, Nagy, Andras, and Yel, Leman

Subjects
antibody deficiency, human, immunodeficiency diseases, immunoglobulins, inborn errors of immunity (IEI), Humans, Hyaluronoglucosaminidase, Male, Europe, Female, Middle Aged, Prospective Studies, Adult, Aged, Immunoglobulins, Injections, Subcutaneous, Immunologic Deficiency Syndromes, Young Adult
Abstract

Aim: To assess the long-term safety of hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 10% in European routine clinical practice.Materials & methods: This prospective, noninterventional, open-label, post-authorization safety study (EUPAS5812) sourced data on adverse events, immunogenicity, treatment regimens and product administration for 106 adult patients prescribed fSCIG 10% across 17 sites in six European countries from July 2014 to February 2020.Results: In total, 1171 treatment-emergent adverse events were reported in 94 patients (88.7%); 25.5% of these events were considered related to fSCIG 10%. Positive binding antibody titers developed in three patients; no neutralizing antibodies to recombinant human hyaluronidase were detected.Conclusion: This real-world study of fSCIG 10% is the longest to date and confirms its long-term safety and tolerability in adults with antibody deficiency diseases.

Published
2024

2. Autoantibodies against type I IFNs in humans with alternative NF-κB pathway deficiency.

Author

Yu, David, Miller, Corey, Feng, Yi, Guichard, Audrey, Béziat, Vivien, Bustamante, Jacinta, Pan-Hammarström, Qiang, Zhang, Yu, Rosen, Lindsey, Holland, Steve, Bosticardo, Marita, Kenney, Heather, Castagnoli, Riccardo, Slade, Charlotte, Boztuğ, Kaan, Mahlaoui, Nizar, Latour, Sylvain, Abraham, Roshini, Lougaris, Vassilios, Hauck, Fabian, Sediva, Anna, Atschekzei, Faranaz, Sogkas, Georgios, Poli, M, Slatter, Mary, Palterer, Boaz, Keller, Michael, Pinzon-Charry, Alberto, Sullivan, Anna, Droney, Luke, Suan, Daniel, Wong, Melanie, Kane, Alisa, Hu, Hannah, Ma, Cindy, Grombiříková, Hana, Ciznar, Peter, Dalal, Ilan, Aladjidi, Nathalie, Hie, Miguel, Lazaro, Estibaliz, Franco, Jose, Keles, Sevgi, Malphettes, Marion, Pasquet, Marlene, Maccari, Maria, Meinhardt, Andrea, Ikinciogullari, Aydan, Shahrooei, Mohammad, Celmeli, Fatih, Frosk, Patrick, Goodnow, Christopher, Gray, Paul, Belot, Alexandre, Kuehn, Hye, Rosenzweig, Sergio, Miyara, Makoto, Licciardi, Francesco, Servettaz, Amélie, Barlogis, Vincent, Le Guenno, Guillaume, Herrmann, Vera-Maria, Kuijpers, Taco, Ducoux, Grégoire, Sarrot-Reynauld, Françoise, Schuetz, Catharina, Cunningham-Rundles, Charlotte, Rieux-Laucat, Frédéric, Tangye, Stuart, Sobacchi, Cristina, Doffinger, Rainer, Warnatz, Klaus, Grimbacher, Bodo, Fieschi, Claire, Berteloot, Laureline, Bryant, Vanessa, Trouillet Assant, Sophie, Su, Helen, Neven, Benedicte, Abel, Laurent, Zhang, Qian, Boisson, Bertrand, Cobat, Aurélie, Jouanguy, Emmanuelle, Kampe, Olle, Bastard, Paul, Roifman, Chaim, Landegren, Nils, Notarangelo, Luigi, Le Voyer, Tom, Parent, Audrey, Liu, Xian, Cederholm, Axel, Gervais, Adrian, Rosain, Jérémie, Nguyen, Tina, Perez Lorenzo, Malena, Rackaityte, Elze, Rinchai, Darawan, and Zhang, Peng

Subjects
Humans, Autoantibodies, COVID-19, Gain of Function Mutation, Genetic Predisposition to Disease, Heterozygote, I-kappa B Proteins, Interferon Type I, Loss of Function Mutation, NF-kappa B, NF-kappa B p52 Subunit, Pneumonia, Viral, Thymus Gland, Thyroid Epithelial Cells
Abstract

Patients with autoimmune polyendocrinopathy syndrome type 1 (APS-1) caused by autosomal recessive AIRE deficiency produce autoantibodies that neutralize type I interferons (IFNs)1,2, conferring a predisposition to life-threatening COVID-19 pneumonia3. Here we report that patients with autosomal recessive NIK or RELB deficiency, or a specific type of autosomal-dominant NF-κB2 deficiency, also have neutralizing autoantibodies against type I IFNs and are at higher risk of getting life-threatening COVID-19 pneumonia. In patients with autosomal-dominant NF-κB2 deficiency, these autoantibodies are found only in individuals who are heterozygous for variants associated with both transcription (p52 activity) loss of function (LOF) due to impaired p100 processing to generate p52, and regulatory (IκBδ activity) gain of function (GOF) due to the accumulation of unprocessed p100, therefore increasing the inhibitory activity of IκBδ (hereafter, p52LOF/IκBδGOF). By contrast, neutralizing autoantibodies against type I IFNs are not found in individuals who are heterozygous for NFKB2 variants causing haploinsufficiency of p100 and p52 (hereafter, p52LOF/IκBδLOF) or gain-of-function of p52 (hereafter, p52GOF/IκBδLOF). In contrast to patients with APS-1, patients with disorders of NIK, RELB or NF-κB2 have very few tissue-specific autoantibodies. However, their thymuses have an abnormal structure, with few AIRE-expressing medullary thymic epithelial cells. Human inborn errors of the alternative NF-κB pathway impair the development of AIRE-expressing medullary thymic epithelial cells, thereby underlying the production of autoantibodies against type I IFNs and predisposition to viral diseases.

Published
2023

4. Autoantibodies against type I IFNs in humans with alternative NF-κB pathway deficiency

Author

Le Voyer, Tom, Parent, Audrey V., Liu, Xian, Cederholm, Axel, Gervais, Adrian, Rosain, Jérémie, Nguyen, Tina, Perez Lorenzo, Malena, Rackaityte, Elze, Rinchai, Darawan, Zhang, Peng, Bizien, Lucy, Hancioglu, Gonca, Ghillani-Dalbin, Pascale, Charuel, Jean-Luc, Philippot, Quentin, Gueye, Mame Sokhna, Maglorius Renkilaraj, Majistor Raj Luxman, Ogishi, Masato, Soudée, Camille, Migaud, Mélanie, Rozenberg, Flore, Momenilandi, Mana, Riller, Quentin, Imberti, Luisa, Delmonte, Ottavia M., Müller, Gabriele, Keller, Baerbel, Orrego, Julio, Franco Gallego, William Alexander, Rubin, Tamar, Emiroglu, Melike, Parvaneh, Nima, Eriksson, Daniel, Aranda-Guillen, Maribel, Berrios, David I., Vong, Linda, Katelaris, Constance H., Mustillo, Peter, Raedler, Johannes, Bohlen, Jonathan, Bengi Celik, Jale, Astudillo, Camila, Winter, Sarah, McLean, Catriona, Guffroy, Aurélien, DeRisi, Joseph L., Yu, David, Miller, Corey, Feng, Yi, Guichard, Audrey, Béziat, Vivien, Bustamante, Jacinta, Pan-Hammarström, Qiang, Zhang, Yu, Rosen, Lindsey B., Holland, Steve M., Bosticardo, Marita, Kenney, Heather, Castagnoli, Riccardo, Slade, Charlotte A., Boztuğ, Kaan, Mahlaoui, Nizar, Latour, Sylvain, Abraham, Roshini S., Lougaris, Vassilios, Hauck, Fabian, Sediva, Anna, Atschekzei, Faranaz, Sogkas, Georgios, Poli, M. Cecilia, Slatter, Mary A., Palterer, Boaz, Keller, Michael D., Pinzon-Charry, Alberto, Sullivan, Anna, Droney, Luke, Suan, Daniel, Wong, Melanie, Kane, Alisa, Hu, Hannah, Ma, Cindy, Grombiříková, Hana, Ciznar, Peter, Dalal, Ilan, Aladjidi, Nathalie, Hie, Miguel, Lazaro, Estibaliz, Franco, Jose, Keles, Sevgi, Malphettes, Marion, Pasquet, Marlene, Maccari, Maria Elena, Meinhardt, Andrea, Ikinciogullari, Aydan, Shahrooei, Mohammad, Celmeli, Fatih, Frosk, Patrick, Goodnow, Christopher C., Gray, Paul E., Belot, Alexandre, Kuehn, Hye Sun, Rosenzweig, Sergio D., Miyara, Makoto, Licciardi, Francesco, Servettaz, Amélie, Barlogis, Vincent, Le Guenno, Guillaume, Herrmann, Vera-Maria, Kuijpers, Taco, Ducoux, Grégoire, Sarrot-Reynauld, Françoise, Schuetz, Catharina, Cunningham-Rundles, Charlotte, Rieux-Laucat, Frédéric, Tangye, Stuart G., Sobacchi, Cristina, Doffinger, Rainer, Warnatz, Klaus, Grimbacher, Bodo, Fieschi, Claire, Berteloot, Laureline, Bryant, Vanessa L., Trouillet Assant, Sophie, Su, Helen, Neven, Benedicte, Abel, Laurent, Zhang, Qian, Boisson, Bertrand, Cobat, Aurélie, Jouanguy, Emmanuelle, Kampe, Olle, Bastard, Paul, Roifman, Chaim M., Landegren, Nils, Notarangelo, Luigi D., Anderson, Mark S., Casanova, Jean-Laurent, and Puel, Anne

Published
2023
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6. Common and Uncommon CT Findings in CVID-Related GL-ILD: Correlations with Clinical Parameters, Therapeutic Decisions and Potential Implications in the Differential Diagnosis

Author

Scarpa, Riccardo, Cinetto, Francesco, Milito, Cinzia, Gianese, Sabrina, Soccodato, Valentina, Buso, Helena, Garzi, Giulia, Carrabba, Maria, Messina, Emanuele, Panebianco, Valeria, Catalano, Carlo, Morana, Giovanni, Lougaris, Vassilios, Landini, Nicholas, and Bondioni, Maria Pia

Published
2023
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7. Hematopoietic stem cell transplantation for CTLA-4 insufficiency across Europe: A European Society for Blood and Marrow Transplantation Inborn Errors Working Party study

Author

Tsilifis, Christo, Speckmann, Carsten, Lum, Su Han, Fox, Thomas A., Soler, Adriana Margarit, Mozo, Yasmina, Corral, Dolores, Ewins, Anna-Maria, Hague, Rosie, Oikonomopoulou, Christina, Kałwak, Krzysztof, Drabko, Katarzyna, Wynn, Robert, Morris, Emma C., Elcombe, Suzanne, Bigley, Venetia, Lougaris, Vassilios, Malagola, Michele, Hauck, Fabian, Sedlacek, Petr, Laberko, Alexandra, Tjon, Jennifer M.L., Buddingh, Emilie P., Wehr, Claudia, Grimbacher, Bodo, Gennery, Andrew R., Lankester, Arjan C., Albert, Michael H., Neven, Bénédicte, and Slatter, Mary A.

Published
2024
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9. Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity

Author

Seidel, Markus G., Seppänen, Mikko R.J., Gennery, Andrew, Kanariou, Maria G., Tantou, Sofia, Grigoriadou, Sofia, Cericola, Gabriella, Hanitsch, Leif G., Scheibenbogen, Carmen, Hlaváčková, Eva O., Krivan, Gergely, McGuire, Frances K., Leahy, Timothy Ronan, Edgar, John David M., Bakhtiar, Shahrzad, Bader, Peter, Rohner, Geraldine Blanchard, Haerynck, Filomeen, Claes, Karlien, Lehmberg, Kai, Müller, Ingo, Farmand, Susan, Fasshauer, Maria, Graf, Dagmar, Neves, Joao Farela, Kostyuchenko, Larysa, Gonzalez-Granado, Luis Ignacio, Jeseňák, Miloš, Carrabba, Maria, Fabio, Giovanna, Pignata, Claudio, Giardino, Giuliana, Karadağ, Ilknur Kökçü, Yıldıran, Alişan, Hancioglu, Gonca, Králíčková, Pavlína, Steinmann, Sandra, Pietrucha, Barbara Maria, Gernert, Michael, Soomann, Maarja, Witte, Torsten, Markocsy, Adam, Wolska-Kusnierz, Beata, Randrianomenjanahary, Philippe, Rouger, Jérémie, Kostaridou, Stavroula, Zabara, Dariia V., Rodina, Yulia A., Shvets, Oksana A., Maccari, Maria Elena, Wolkewitz, Martin, Schwab, Charlotte, Lorenzini, Tiziana, Leiding, Jennifer W., Aladjdi, Nathalie, Abolhassani, Hassan, Abou-Chahla, Wadih, Aiuti, Alessandro, Azarnoush, Saba, Baris, Safa, Barlogis, Vincent, Barzaghi, Federica, Baumann, Ulrich, Bloomfield, Marketa, Bohynikova, Nadezda, Bodet, Damien, Boutboul, David, Bucciol, Giorgia, Buckland, Matthew S., Burns, Siobhan O., Cancrini, Caterina, Cathébras, Pascal, Cavazzana, Marina, Cheminant, Morgane, Chinello, Matteo, Ciznar, Peter, Coulter, Tanya I., D’Aveni, Maud, Ekwall, Olov, Eric, Zelimir, Eren, Efrem, Fasth, Anders, Frange, Pierre, Fournier, Benjamin, Garcia-Prat, Marina, Gardembas, Martine, Geier, Christoph, Ghosh, Sujal, Goda, Vera, Hammarström, Lennart, Hauck, Fabian, Heeg, Maximilian, Heropolitanska-Pliszka, Edyta, Hilfanova, Anna, Jolles, Stephen, Karakoc-Aydiner, Elif, Kindle, Gerhard R., Kiykim, Ayca, Klemann, Christian, Koletsi, Patra, Koltan, Sylwia, Kondratenko, Irina, Körholz, Julia, Krüger, Renate, Jeziorski, Eric, Levy, Romain, Le Guenno, Guillaume, Lefevre, Guillaume, Lougaris, Vassilios, Marzollo, Antonio, Mahlaoui, Nizar, Malphettes, Marion, Meinhardt, Andrea, Merlin, Etienne, Meyts, Isabelle, Milota, Tomas, Moreira, Fernando, Moshous, Despina, Mukhina, Anna, Neth, Olaf, Neubert, Jennifer, Neven, Benedicte, Nieters, Alexandra, Nove-Josserand, Raphaele, Oksenhendler, Eric, Ozen, Ahmet, Olbrich, Peter, Perlat, Antoinette, Pac, Malgorzata, Schmid, Jana Pachlopnik, Pacillo, Lucia, Parra-Martinez, Alba, Paschenko, Olga, Pellier, Isabelle, Sefer, Asena Pinar, Plebani, Alessandro, Plantaz, Dominique, Prader, Seraina, Raffray, Loic, Ritterbusch, Henrike, Riviere, Jacques G., Rivalta, Beatrice, Rusch, Stephan, Sakovich, Inga, Savic, Sinisa, Scheible, Raphael, Schleinitz, Nicolas, Schuetz, Catharina, Schulz, Ansgar, Sediva, Anna, Semeraro, Michaela, Sharapova, Svetlana O., Shcherbina, Anna, Slatter, Mary A., Sogkas, Georgios, Soler-Palacin, Pere, Speckmann, Carsten, Stephan, Jean-Louis, Suarez, Felipe, Tommasini, Alberto, Trück, Johannes, Uhlmann, Annette, van Aerde, Koen J., van Montfrans, Joris, von Bernuth, Horst, Warnatz, Klaus, Williams, Tony, Worth, Austen J.J., Ip, Winnie, Picard, Capucine, Catherinot, Emilie, Nademi, Zohreh, Grimbacher, Bodo, Forbes Satter, Lisa R., Kracker, Sven, Chandra, Anita, Condliffe, Alison M., and Ehl, Stephan

Published
2023
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10. Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo

Author

Asperti, Claudia, Canarutto, Daniele, Porcellini, Simona, Sanvito, Francesca, Cecere, Francesca, Vavassori, Valentina, Ferrari, Samuele, Rovelli, Elisabetta, Albano, Luisa, Jacob, Aurelien, Sergi Sergi, Lucia, Montaldo, Elisa, Ferrua, Francesca, González-Granado, Luis Ignacio, Lougaris, Vassilios, Badolato, Raffaele, Finocchi, Andrea, Villa, Anna, Radrizzani, Marina, and Naldini, Luigi

Published
2023
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12. Coronavirus disease 2019 in patients with inborn errors of immunity: An international study

Author

Meyts, Isabelle, Bucciol, Giorgia, Quinti, Isabella, Neven, Bénédicte, Fischer, Alain, Seoane, Elena, Lopez-Granados, Eduardo, Gianelli, Carla, Robles-Marhuenda, Angel, Jeandel, Pierre-Yves, Paillard, Catherine, Sankaran, Vijay G, Demirdag, Yesim Yilmaz, Lougaris, Vassilios, Aiuti, Alessandro, Plebani, Alessandro, Milito, Cinzia, Dalm, Virgil ASH, Guevara-Hoyer, Kissy, Sánchez-Ramón, Silvia, Bezrodnik, Liliana, Barzaghi, Federica, Gonzalez-Granado, Luis Ignacio, Hayman, Grant R, Uzel, Gulbu, Mendonça, Leonardo Oliveira, Agostini, Carlo, Spadaro, Giuseppe, Badolato, Raffaele, Soresina, Annarosa, Vermeulen, François, Bosteels, Cedric, Lambrecht, Bart N, Keller, Michael, Mustillo, Peter J, Abraham, Roshini S, Gupta, Sudhir, Ozen, Ahmet, Karakoc-Aydiner, Elif, Baris, Safa, Freeman, Alexandra F, Yamazaki-Nakashimada, Marco, Scheffler-Mendoza, Selma, Espinosa-Padilla, Sara, Gennery, Andrew R, Jolles, Stephen, Espinosa, Yazmin, Poli, M Cecilia, Fieschi, Claire, Hauck, Fabian, Cunningham-Rundles, Charlotte, Mahlaoui, Nizar, Errors of Immunity, IUIS Committee of Inborn, Warnatz, Klaus, Sullivan, Kathleen E, and Tangye, Stuart G

Subjects
Vaccine Related, Clinical Research, Pneumonia & Influenza, Prevention, Pediatric, Emerging Infectious Diseases, Pneumonia, Infectious Diseases, Lung, Biodefense, Rare Diseases, Good Health and Well Being, Adolescent, Adult, Aged, COVID-19, Child, Child, Preschool, Female, Genetic Diseases, Inborn, Humans, Immunologic Deficiency Syndromes, Infant, Infant, Newborn, Male, Middle Aged, Retrospective Studies, Risk Factors, SARS-CoV-2, Severity of Illness Index, Young Adult, primary immunodeficiencies, inborn errors of immunity, hypogammaglobulinemia, immune dysregulation, IUIS Committee of Inborn Errors of Immunity, Immunology, Allergy
Abstract

BackgroundThere is uncertainty about the impact of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection in individuals with rare inborn errors of immunity (IEI), a population at risk of developing severe coronavirus disease 2019. This is relevant not only for these patients but also for the general population, because studies of IEIs can unveil key requirements for host defense.ObjectiveWe sought to describe the presentation, manifestations, and outcome of SARS-CoV-2 infection in IEI to inform physicians and enhance understanding of host defense against SARS-CoV-2.MethodsAn invitation to participate in a retrospective study was distributed globally to scientific, medical, and patient societies involved in the care and advocacy for patients with IEI.ResultsWe gathered information on 94 patients with IEI with SARS-CoV-2 infection. Their median age was 25 to 34 years. Fifty-three patients (56%) suffered from primary antibody deficiency, 9 (9.6%) had immune dysregulation syndrome, 6 (6.4%) a phagocyte defect, 7 (7.4%) an autoinflammatory disorder, 14 (15%) a combined immunodeficiency, 3 (3%) an innate immune defect, and 2 (2%) bone marrow failure. Ten were asymptomatic, 25 were treated as outpatients, 28 required admission without intensive care or ventilation, 13 required noninvasive ventilation or oxygen administration, 18 were admitted to intensive care units, 12 required invasive ventilation, and 3 required extracorporeal membrane oxygenation. Nine patients (7 adults and 2 children) died.ConclusionsThis study demonstrates that (1) more than 30% of patients with IEI had mild coronavirus disease 2019 (COVID-19) and (2) risk factors predisposing to severe disease/mortality in the general population also seemed to affect patients with IEI, including more younger patients. Further studies will identify pathways that are associated with increased risk of severe disease and are nonredundant or redundant for protection against SARS-CoV-2.

Published
2021

14. Immune tolerance breakdown in inborn errors of immunity: Paving the way to novel therapeutic approaches

Author

Giardino, Giuliana, Romano, Roberta, Lougaris, Vassilios, Castagnoli, Riccardo, Cillo, Francesca, Leonardi, Lucia, La Torre, Francesco, Soresina, Annarosa, Federici, Silvia, Cancrini, Caterina, Pacillo, Lucia, Toriello, Elisabetta, Cinicola, Bianca Laura, Corrente, Stefania, Volpi, Stefano, Marseglia, Gian Luigi, Pignata, Claudio, and Cardinale, Fabio

Published
2023
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15. A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome

Author

Rao, V. Koneti, Webster, Sharon, Šedivá, Anna, Plebani, Alessandro, Schuetz, Catharina, Shcherbina, Anna, Conlon, Niall, Coulter, Tanya, Dalm, Virgil A., Trizzino, Antonino, Zharankova, Yulia, Kulm, Elaine, Körholz, Julia, Lougaris, Vassilios, Rodina, Yulia, Radford, Kath, Bradt, Jason, Kucher, Klaus, Relan, Anurag, Holland, Steven M., Lenardo, Michael J., and Uzel, Gulbu

Published
2023
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16. The Impact of SARS-CoV-2 Infection in Patients with Inborn Errors of Immunity: the Experience of the Italian Primary Immunodeficiencies Network (IPINet)

Author

Giardino, Giuliana, Milito, Cinzia, Lougaris, Vassilios, Punziano, Alessandra, Carrabba, Maria, Cinetto, Francesco, Scarpa, Riccardo, Dellepiane, Rosa Maria, Ricci, Silvia, Rivalta, Beatrice, Conti, Francesca, Marzollo, Antonio, Firinu, Davide, Cirillo, Emilia, Lagnese, Gianluca, Cancrini, Caterina, Martire, Baldassare, Danieli, Maria Giovanna, Pession, Andrea, Vacca, Angelo, Azzari, Chiara, Fabio, Giovanna, Soresina, Annarosa, Agostini, Carlo, Spadaro, Giuseppe, Badolato, Raffaele, Cicalese, Maria Pia, Aiuti, Alessandro, Plebani, Alessandro, Quinti, Isabella, and Pignata, Claudio

Published
2022
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18. Progressive Depletion of B and T Lymphocytes in Patients with Ataxia Telangiectasia: Results of the Italian Primary Immunodeficiency Network

Author

Cirillo, Emilia, Polizzi, Agata, Soresina, Annarosa, Prencipe, Rosaria, Giardino, Giuliana, Cancrini, Caterina, Finocchi, Andrea, Rivalta, Beatrice, Dellepiane, Rosa M., Baselli, Lucia A., Montin, Davide, Trizzino, Antonino, Consolini, Rita, Azzari, Chiara, Ricci, Silvia, Lodi, Lorenzo, Quinti, Isabella, Milito, Cinzia, Leonardi, Lucia, Duse, Marzia, Carrabba, Maria, Fabio, Giovanna, Bertolini, Patrizia, Coccia, Paola, D’Alba, Irene, Pession, Andrea, Conti, Francesca, Zecca, Marco, Lunardi, Claudio, Bianco, Manuela Lo, Presti, Santiago, Sciuto, Laura, Micheli, Roberto, Bruzzese, Dario, Lougaris, Vassilios, Badolato, Raffaele, Plebani, Alessandro, Chessa, Luciana, and Pignata, Claudio

Published
2022
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20. Immunological Evaluation of Patients Affected with Jacobsen Syndrome Reveals Profound Not Age-Related Lymphocyte Alterations

Author

Baronio, Manuela, Saettini, Francesco, Gazzurelli, Luisa, Rossi, Stefano, Marzollo, Antonio, Ricci, Silvia, Zama, Daniele, Palterer, Boaz, Clementina, Canessa, Lorenzo, Lodi, Chiarini, Marco, Sottini, Alessandra, Imberti, Luisa, Gorio, Chiara, Rossini, Linda, Badolato, Raffaele, Plebani, Alessandro, Moratto, Daniele, and Lougaris, Vassilios

Published
2022
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22. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

Author

Boztug, Kaan, Brunner, Juergen, Demel, Ulrike F., Förster-Waldl, Elisabeth, Gasteiger, Lukas M., Göschl, Lisa, Kojić, Marina, Schroll, Andrea, Seidel, Markus G., Wintergerst, Uwe, Wisgrill, Lukas, Sharapova, Svetlana O., Goffard, Jean-Christophe, Kerre, Tessa, Meyts, Isabelle, Roosens, Fine, Smet, Julie, Haerynck, Filomeen, Eric, Zelimir Pavle, Milenova, Veneta, Gagro, Alenka, Richter, Darko, Chovancova, Zita, Hlavackova, Eva, Litzman, Jiri, Milota, Tomas, Sediva, Anna, Elaziz, Dalia Abd, Alkady, Radwa Salaheldin, El Sayed El Hawary, Rabab, Eldash, Alia S., Galal, Nermeen, Lotfy, Sohilla, Meshaal, Safa S., Reda, Shereen M., Sobh, Ali, Elmarsafy, Aisha, Seppänen, Mikko R.J., Brosselin, Pauline, Courteille, Virginie, De Vergnes, Nathalie, Kracker, Sven, Pergent, Martine, Randrianomenjanahary, Philippe, Ahrenstorf, Gerrit, Albert, Michael H., Ankermann, Tobias, Atschekzei, Faranaz, Baumann, Ulrich, Becker, Benjamin C., Behrends, Uta, Belohradsky, Bernd H., Biegner, Anika-Kerstin, Binder, Nadine, Bode, Sebastian F.N., Boesecke, Christoph, Boetticher, Benedikt, Borte, Michael, Borte, Stephan, Classen, Carl Friedrich, Dirks, Johannes, Dückers, Gregor, El-Helou, Sabine, Ernst, Diana, Fasshauer, Maria, Fecker, Gisela, Felgentreff, Kerstin, Foell, Dirk, Ghosh, Sujal, Girschick, Hermann J., Goldacker, Sigune, Graf, Norbert, Graf, Dagmar, Greil, Johann, Hanitsch, Leif Gunnar, Hauck, Fabian, Heeg, Maximilian, Heine, Sabine I., Henes, Joerg C., Hoenig, Manfred, Holzer, Ursula, Holzinger, Dirk, Horneff, Gerd, Hundsdoerfer, Patrick, Jablonka, Alexandra, Jakoby, Donate, Joean, Oana, Kaiser-Labusch, Petra, Klemann, Christian, Kobbe, Robin, Körholz, Julia, Kramm, Christof M., Krüger, Renate, Landwehr-Kenzel, Sybille, Lehmberg, Kai, Liese, Johannes G., Lippert, Conrad Ferdinand, Maccari, Maria Elena, Masjosthusmann, Katja, Meinhardt, Andrea, Metzler, Markus, Morbach, Henner, Müller, Ingo, Naumann-Bartsch, Nora, Neubert, Jennifer, Niehues, Tim, Peter, Hans-Hartmut, Rieber, Nikolaus, Ritterbusch, Henrike, Rockstroh, Jürgen Kurt, Roesler, Joachim, Schauer, Uwe, Scheible, Raphael, Schmalzing, Marc, Schmidt, Reinhold Ernst, Schneider, Dominik T., Schreiber, Stefan, Schuetz, Catharina, Schulz, Ansgar, Schulze-Koops, Hendrik, Schulze-Sturm, Ulf, Schuster, Volker, Schwaneck, Eva C., Schwarz, Klaus, Schwarze-Zander, Carolynne, Sirin, Mehtap, Skapenko, Alla, Sogkas, Georgios, Sparber-Sauer, Monika, Speckmann, Carsten, Steinmann, Sandra, Stiehler, Sophie, Tenbrock, Klaus, von Bernuth, Horst, Warnatz, Klaus, Wasmuth, Jan-Christian, Weiss, Michael, Witte, Torsten, Wittke, Kirsten, Wittkowski, Helmut, Zeuner, Rainald A., Farmaki, Evangelia, Hatzistilianou, Maria N., Kakkas, Ioannis, Kanariou, Maria G., Kapousouzi, Androniki, Liatsis, Emmanouil, Maggina, Paraskevi, Papadopoulou-Alataki, Efimia, Raptaki, Maria, Speletas, Matthaios, Tantou, Sofia, Goda, Vera, Kriván, Gergely, Marodi, Laszlo, Abolhassani, Hassan, Aghamohammadi, Asghar, Rezaei, Nima, Feighery, Conleth, Leahy, Timothy Ronan, Ryan, Paul, Batzir, Nurit Assia, Garty, Ben Zion, Tamary, Hannah, Aiuti, Alessandro, Amodio, Donato, Azzari, Chiara, Barzaghi, Federica, Baselli, Lucia A., Cancrini, Caterina, Carrabba, Maria, Cazzaniga, Marco, Cesaro, Simone, Chinello, Matteo, Danieli, Maria Giovanna, Dellepiane, Rosa Maria, Fabio, Giovanna, Gambineri, Eleonora, Lodi, Lorenzo, Lougaris, Vassilios, Marasco, Carolina, Martire, Baldassarre, Marzollo, Antonio, Milito, Cinzia, Moschese, Viviana, Pignata, Claudio, Plebani, Alessandro, Porta, Fulvio, Quinti, Isabella, Ricci, Silvia, Soresina, Annarosa, Tommasini, Alberto, Vacca, Angelo, Vanessa, Clementina, Blažienė, Audra, Sitkauskiene, Brigita, Gowin, Ewelina, Heropolitańska-Pliszka, Edyta, Pietrucha, Barbara, Szaflarska, Anna, Więsik-Szewczyk, Ewa, Wolska-Kuśnierz, Beata, Esteves, Isabel, Faria, Emilia, Marques, Laura Hora, Neves, João Farela, Silva, Susana L., Teixeira, Carla, Pereira da Silva, Sara, Capilna, Brindusa Ruxandra, Guseva, Marina N., Shcherbina, Anna, Bobcakova, Anna, Ciznar, Peter, Gabzdilova, Juliana, Jesenak, Milos, Kapustova, Lenka, Orosova, Jaroslava, Petrovicova, Otilia, Raffac, Stefan, Kopač, Peter, Allende, Luis M., Antolí, Arnau, Blanch, Gemma Rocamora, Carbone, Javier, Dieli-Crimi, Romina, Garcia-Prat, Marina, Gil-Herrera, Juana, Gonzalez-Granado, Luis Ignacio, Agulló, Pilar Llobet, Olbrich, Peter, Parra-Martínez, Alba, Paz-Artal, Estela, Pleguezuelo, Daniel E., Rodríguez, Nerea Salmón, Sánchez-Ramón, Silvia, Santos-Pérez, Juan Luis, Solanich, Xavier, Soler-Palacin, Pere, González-Amores, Miriam, Ekwall, Olov, Fasth, Anders, Bitzenhofer-Grüber, Michaela, Candotti, Fabio, Dimitriou, Florentia, Heininger, Ulrich, Holbro, Andreas, Jandus, Peter, Kolios, Antonios G.A., Marschall, Karin, Schmid, Jana Pachlopnik, Posfay-Barbe, Klara M., Prader, Seraina, Reichenbach, Janine, Steiner, Urs C., Trück, Johannes, Bredius, Robbert G., de Kruijf- Bazen, Suzanne, de Vries, Esther, Henriet, Stefanie S.V., Kuijpers, Taco W., Potjewijd, Judith, Rutgers, Abraham, Stol, Kim, van Aerde, Koen J., Van den Berg, J. Merlijn, van de Ven, Annick A.J.M., Montfrans, Jorisvan, Aydemir, Sezin, Baris, Safa, Dogu, Figen, Ikinciogullari, Aydan, Karakoc-Aydiner, Elif, Kilic, Sara S., Kiykim, Ayca, Kökçü Karadağ, Şefika İlknur, Kutukculer, Necil, Ocak, Suheyla, UNAL, Ekrem, Boyarchuk, Oksana, Hilfanova, Anna, Kostyuchenko, Larysa V., Alachkar, Hana, Arkwright, Peter D., Baxendale, Helen E., Bernatoniene, Jolanta, Coulter, Tanya I., Garcez, Tomaz, Goddard, Sarah, Gompels, Mark M., Grigoriadou, Sofia, Herriot, Richard, Herwadkar, Archana, Huissoon, Aarnoud, Ibberson, Lisa, Nademi, Zoreh, Noorani, Sadia, Parvin, Shahnaz, Steele, Cathal Laurence, Thomas, Moira, Waruiru, Catherine, Yong, Patrick F.K., Bourne, Helen, Thalhammer, Julian, Kindle, Gerhard, Nieters, Alexandra, Rusch, Stephan, Fischer, Alain, Grimbacher, Bodo, Edgar, David, Buckland, Matthew, Mahlaoui, Nizar, and Ehl, Stephan

Published
2021
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24. Corrigendum: Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56bright NKG2A+++ Cells, and Yet Display Increased Degranulation and Higher Perforin Content

Author

Dobbs, Kerry, Tabellini, Giovanna, Calzoni, Enrica, Patrizi, Ornella, Martinez, Paula, Giliani, Silvia Clara, Moratto, Daniele, Al-Herz, Waleed, Cancrini, Caterina, Cowan, Morton, Bleesing, Jacob, Booth, Claire, Buchbinder, David, Burns, Siobhan O, Chatila, Talal A, Chou, Janet, Daza-Cajigal, Vanessa, de Bruin, Lisa M Ott, de la Morena, Maite Teresa, Di Matteo, Gigliola, Finocchi, Andrea, Geha, Raif, Goyal, Rakesh K, Hayward, Anthony, Holland, Steven, Huang, Chiung-Hui, Kanariou, Maria G, King, Alejandra, Kaplan, Blanka, Kleva, Anastasiya, Kuijpers, Taco W, Lee, Bee Wah, Lougaris, Vassilios, Massaad, Michel, Meyts, Isabelle, Morsheimer, Megan, Neven, Benedicte, Pai, Sung-Yun, Parvaneh, Nima, Plebani, Alessandro, Prockop, Susan, Reisli, Ismail, Soh, Jian Yi, Somech, Raz, Torgerson, Troy R, Kim, Yae-Jean, Walter, Jolan E, Gennery, Andrew R, Keles, Sevgi, Manis, John P, Marcenaro, Emanuela, Moretta, Alessandro, Parolini, Silvia, and Notarangelo, Luigi D

Subjects
natural killer cells, recombinase-activating genes, non-homologous end joining, immunodeficiency, CD56, interferon-gamma, degranulation, interferon-γ, Immunology, Medical Microbiology
Abstract

[This corrects the article on p. 798 in vol. 8, PMID: 28769923.].

Published
2017

25. Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56bright NKG2A+++ Cells, and Yet Display Increased Degranulation and Higher Perforin Content

Author

Dobbs, Kerry, Tabellini, Giovanna, Calzoni, Enrica, Patrizi, Ornella, Martinez, Paula, Giliani, Silvia Clara, Moratto, Daniele, Al-Herz, Waleed, Cancrini, Caterina, Cowan, Morton, Bleesing, Jacob, Booth, Claire, Buchbinder, David, Burns, Siobhan O, Chatila, Talal A, Chou, Janet, Daza-Cajigal, Vanessa, de Bruin, Lisa M Ott, de la Morena, Maite Teresa, Di Matteo, Gigliola, Finocchi, Andrea, Geha, Raif, Goyal, Rakesh K, Hayward, Anthony, Holland, Steven, Huang, Chiung-Hui, Kanariou, Maria G, King, Alejandra, Kaplan, Blanka, Kleva, Anastasiya, Kuijpers, Taco W, Lee, Bee Wah, Lougaris, Vassilios, Massaad, Michel, Meyts, Isabelle, Morsheimer, Megan, Neven, Benedicte, Pai, Sung-Yun, Parvaneh, Nima, Plebani, Alessandro, Prockop, Susan, Reisli, Ismail, Soh, Jian Yi, Somech, Raz, Torgerson, Troy R, Kim, Yae-Jaen, Walter, Jolan E, Gennery, Andrew R, Keles, Sevgi, Manis, John P, Marcenaro, Emanuela, Moretta, Alessandro, Parolini, Silvia, and Notarangelo, Luigi D

Subjects
Genetics, Rare Diseases, Stem Cell Research, Regenerative Medicine, Transplantation, Stem Cell Research - Nonembryonic - Human, natural killer cells, recombinase-activating genes, non-homologous end joining, immunodeficiency, CD56, interferon-gamma, degranulation, interferon-γ, Immunology, Medical Microbiology
Abstract

Mutations of the recombinase-activating genes 1 and 2 (RAG1 and RAG2) in humans are associated with a broad range of phenotypes. For patients with severe clinical presentation, hematopoietic stem cell transplantation (HSCT) represents the only curative treatment; however, high rates of graft failure and incomplete immune reconstitution have been observed, especially after unconditioned haploidentical transplantation. Studies in mice have shown that Rag-/- natural killer (NK) cells have a mature phenotype, reduced fitness, and increased cytotoxicity. We aimed to analyze NK cell phenotype and function in patients with mutations in RAG and in non-homologous end joining (NHEJ) genes. Here, we provide evidence that NK cells from these patients have an immature phenotype, with significant expansion of CD56bright CD16-/int CD57- cells, yet increased degranulation and high perforin content. Correlation was observed between in vitro recombinase activity of the mutant proteins, NK cell abnormalities, and in vivo clinical phenotype. Addition of serotherapy in the conditioning regimen, with the aim of depleting the autologous NK cell compartment, may be important to facilitate engraftment and immune reconstitution in patients with RAG and NHEJ defects treated by HSCT.

Published
2017

27. Long-term safety of hyaluronidase-facilitated subcutaneous immunoglobulin 10%: a European post-authorization study

Author

MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Ellerbroek, Pauline M, Hanitsch, Leif G, Witte, Torsten, Lougaris, Vassilios, Hagen, P Martin van, Paassen, Pieter van, Chen, Jie, Fielhauer, Katharina, McCoy, Barbara, Nagy, Andras, Yel, Leman, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Ellerbroek, Pauline M, Hanitsch, Leif G, Witte, Torsten, Lougaris, Vassilios, Hagen, P Martin van, Paassen, Pieter van, Chen, Jie, Fielhauer, Katharina, McCoy, Barbara, Nagy, Andras, and Yel, Leman

Published
2024

28. λ5 Deficiency

Author

Lougaris, Vassilios, Plebani, Alessandro, Warnatz, Klaus, Section editor, van Montfrans, Joris M., Section editor, Orange, Jordan Scott, editor, Chinen, Javier, editor, MacKay, Ian R., Series Editor, and Rose, Noel R., Series Editor

Published
2020
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29. X-linked Agammaglobulinemia (BTK Deficiency)

Author

Lougaris, Vassilios, Plebani, Alessandro, Warnatz, Klaus, Section editor, van Montfrans, Joris M., Section editor, Orange, Jordan Scott, editor, Chinen, Javier, editor, MacKay, Ian R., Series Editor, and Rose, Noel R., Series Editor

Published
2020
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30. μ Heavy Chain Deficiency

Author

Lougaris, Vassilios, Plebani, Alessandro, Warnatz, Klaus, Section editor, van Montfrans, Joris M., Section editor, Orange, Jordan Scott, editor, Chinen, Javier, editor, MacKay, Ian R., Series Editor, and Rose, Noel R., Series Editor

Published
2020
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32. PIK3R1 Deficiency-Associated Agammaglobulinemia

Author

Lougaris, Vassilios, Plebani, Alessandro, Warnatz, Klaus, Section editor, van Montfrans, Joris M., Section editor, Orange, Jordan Scott, editor, Chinen, Javier, editor, MacKay, Ian R., Series Editor, and Rose, Noel R., Series Editor

Published
2020
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33. Long-term follow-up of 168 patients with X-linked agammaglobulinemia reveals increased morbidity and mortality

Author

Lougaris, Vassilios, Soresina, Annarosa, Baronio, Manuela, Montin, Davide, Martino, Silvana, Signa, Sara, Volpi, Stefano, Zecca, Marco, Marinoni, Maddalena, Baselli, Lucia Augusta, Dellepiane, Rosa Maria, Carrabba, Maria, Fabio, Giovanna, Putti, Maria Caterina, Cinetto, Francesco, Lunardi, Claudio, Gazzurelli, Luisa, Benvenuto, Alessio, Bertolini, Patrizia, Conti, Francesca, Consolini, Rita, Ricci, Silvia, Azzari, Chiara, Leonardi, Lucia, Duse, Marzia, Pulvirenti, Federica, Milito, Cinzia, Quinti, Isabella, Cancrini, Caterina, Finocchi, Andrea, Moschese, Viviana, Cirillo, Emilia, Crescenzi, Ludovica, Spadaro, Giuseppe, Marasco, Carolina, Vacca, Angelo, Cardinale, Fabio, Martire, Baldassare, Trizzino, Antonino, Licciardello, Maria, Cossu, Fausto, Di Matteo, Gigliola, Badolato, Raffaele, Ferrari, Simona, Giliani, Silvia, Pession, Andrea, Ugazio, Alberto, Pignata, Claudio, and Plebani, Alessandro

Published
2020
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34. Vasculitis and vasculopathy associated with inborn errors of immunity: an overview

Author

Federici, Silvia, primary, Cinicola, Bianca Laura, additional, La Torre, Francesco, additional, Castagnoli, Riccardo, additional, Lougaris, Vassilios, additional, Giardino, Giuliana, additional, Volpi, Stefano, additional, Caorsi, Roberta, additional, Leonardi, Lucia, additional, Corrente, Stefania, additional, Soresina, Annarosa, additional, Cancrini, Caterina, additional, Insalaco, Antonella, additional, Gattorno, Marco, additional, De Benedetti, Fabrizio, additional, Marseglia, Gian Luigi, additional, Del Giudice, Michele Miraglia, additional, and Cardinale, Fabio, additional

Published
2024
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35. The Italian Registry for Primary Immunodeficiencies (Italian Primary Immunodeficiency Network; IPINet): Twenty Years of Experience (1999–2019)

Author

Lougaris, Vassilios, Pession, Andrea, Baronio, Manuela, Soresina, Annarosa, Rondelli, Roberto, Gazzurelli, Luisa, Benvenuto, Alessio, Martino, Silvana, Gattorno, Marco, Biondi, Andrea, Zecca, Marco, Marinoni, Maddalena, Fabio, Giovanna, Aiuti, Alessandro, Marseglia, Gianluigi, Putti, Maria Caterina, Agostini, Carlo, Lunardi, Claudio, Tommasini, Alberto, Bertolini, Patrizia, Gambineri, Eleonora, Consolini, Rita, Matucci, Andrea, Azzari, Chiara, Danieli, Maria Giovanna, Paganelli, Roberto, Duse, Marzia, Cancrini, Caterina, Moschese, Viviana, Chessa, Luciana, Spadaro, Giuseppe, Civino, Adele, Vacca, Angelo, Cardinale, Fabio, Martire, Baldassare, Carpino, Luigi, Trizzino, Antonino, Russo, Giovanna, Cossu, Fausto, Badolato, Raffaele, Pietrogrande, Maria Cristina, Quinti, Isabella, Rossi, Paolo, Ugazio, Alberto, Pignata, Claudio, and Plebani, Alessandro

Published
2020
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36. Prevalence of Immunological Defects in a Cohort of 97 Rubinstein–Taybi Syndrome Patients

Author

Saettini, Francesco, Herriot, Richard, Prada, Elisabetta, Nizon, Mathilde, Zama, Daniele, Marzollo, Antonio, Romaniouk, Igor, Lougaris, Vassilios, Cortesi, Manuela, Morreale, Alessia, Kosaki, Rika, Cardinale, Fabio, Ricci, Silvia, Domínguez-Garrido, Elena, Montin, Davide, Vincent, Marie, Milani, Donatella, Biondi, Andrea, Gervasini, Cristina, and Badolato, Raffaele

Published
2020
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39. Double-blind, placebo-controlled, randomized trial on low-dose azithromycin prophylaxis in patients with primary antibody deficiencies

Author

Milito, Cinzia, Pulvirenti, Federica, Cinetto, Francesco, Lougaris, Vassilios, Soresina, Annarosa, Pecoraro, Antonio, Vultaggio, Alessandra, Carrabba, Maria, Lassandro, Giuseppe, Plebani, Alessandro, Spadaro, Giuseppe, Matucci, Andrea, Fabio, Giovanna, Dellepiane, Rosa Maria, Martire, Baldassarre, Agostini, Carlo, Abeni, Damiano, Tabolli, Stefano, and Quinti, Isabella

Published
2019
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40. Long-Term Outcome of WHIM Syndrome in 18 Patients: High Risk of Lung Disease and HPV-Related Malignancies

Author

Dotta, Laura, Notarangelo, Lucia Dora, Moratto, Daniele, Kumar, Rajesh, Porta, Fulvio, Soresina, Annarosa, Lougaris, Vassilios, Plebani, Alessandro, Smith, C. I. Edvard, Norlin, Anna-Carin, Gòmez Raccio, Andrea Cecilia, Bubanska, Eva, Bertolini, Patrizia, Amendola, Giovanni, Visentini, Marcella, Fiorilli, Massimo, Venuti, Aldo, and Badolato, Raffaele

Published
2019
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41. Comparison of Common Monogenic Defects in a Large Predominantly Antibody Deficiency Cohort

Author

Yazdani, Reza, Abolhassani, Hassan, Kiaee, Fatemeh, Habibi, Sima, Azizi, Gholamreza, Tavakol, Marzieh, Chavoshzadeh, Zahra, Mahdaviani, Seyed Alireza, Momen, Tooba, Gharagozlou, Mohammad, Movahedi, Masoud, Hamidieh, Amir Ali, Behniafard, Nasrin, Nabavi, Mohammamd, Bemanian, Mohammad Hassan, Arshi, Saba, Molatefi, Rasol, Sherkat, Roya, Shirkani, Afshin, Amin, Reza, Aleyasin, Soheila, Faridhosseini, Reza, Jabbari-Azad, Farahzad, Mohammadzadeh, Iraj, Ghaffari, Javad, Shafiei, Alireza, Kalantari, Arash, Mansouri, Mahboubeh, Mesdaghi, Mehrnaz, Babaie, Delara, Ahanchian, Hamid, Khoshkhui, Maryam, Soheili, Habib, Eslamian, Mohammad Hossein, Cheraghi, Taher, Dabbaghzadeh, Abbas, Tavassoli, Mahmoud, Kalmarzi, Rasoul Nasiri, Mortazavi, Seyed Hamidreza, Kashef, Sara, Esmaeilzadeh, Hossein, Tafaroji, Javad, Khalili, Abbas, Zandieh, Fariborz, Sadeghi-Shabestari, Mahnaz, Darougar, Sepideh, Behmanesh, Fatemeh, Akbari, Hedayat, Zandkarimi, Mohammadreza, Abolnezhadian, Farhad, Fayezi, Abbas, Moghtaderi, Mojgan, Ahmadiafshar, Akefeh, Shakerian, Behzad, Sajedi, Vahid, Taghvaei, Behrang, Safari, Mojgan, Heidarzadeh, Marzieh, Ghalebaghi, Babak, Fathi, Seyed Mohammad, Darabi, Behzad, Bazregari, Saeed, Bazargan, Nasrin, Fallahpour, Morteza, Khayatzadeh, Alireza, Javahertrash, Naser, Bashardoust, Bahram, Zamani, Mohammadali, Mohsenzadeh, Azam, Ebrahimi, Sarehsadat, Sharafian, Samin, Vosughimotlagh, Ahmad, Tafakoridelbari, Mitra, Rahim, Maziar, Ashournia, Parisa, Razaghian, Anahita, Rezaei, Arezou, Samavat, Ashraf, Mamishi, Setareh, Khazaei, Hossein Ali, Mohammadi, Javad, Negahdari, Babak, Parvaneh, Nima, Rezaei, Nima, Lougaris, Vassilios, Giliani, Silvia, Plebani, Alessandro, Ochs, Hans D., Hammarström, Lennart, and Aghamohammadi, Asghar

Published
2019
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42. Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T‐cells for the treatment of Hyper IgM1

Author

Canarutto, Daniele, primary, Asperti, Claudia, additional, Vavassori, Valentina, additional, Porcellini, Simona, additional, Rovelli, Elisabetta, additional, Paulis, Marianna, additional, Ferrari, Samuele, additional, Varesi, Angelica, additional, Fiumara, Martina, additional, Jacob, Aurelien, additional, Sergi Sergi, Lucia, additional, Visigalli, Ilaria, additional, Ferrua, Francesca, additional, González‐Granado, Luis Ignacio, additional, Lougaris, Vassilios, additional, Finocchi, Andrea, additional, Villa, Anna, additional, Radrizzani, Marina, additional, and Naldini, Luigi, additional

Published
2023
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45. Interim analysis: Open-label extension study of leniolisib for patients with APDS

Author

Rao, V. Koneti, primary, Kulm, Elaine, additional, Šedivá, Anna, additional, Plebani, Alessandro, additional, Schuetz, Catharina, additional, Shcherbina, Anna, additional, Dalm, Virgil A., additional, Trizzino, Antonino, additional, Zharankova, Yulia, additional, Webster, Sharon, additional, Orpia, Alanvin, additional, Körholz, Julia, additional, Lougaris, Vassilios, additional, Rodina, Yulia, additional, Radford, Kath, additional, Bradt, Jason, additional, Relan, Anurag, additional, Holland, Steven M., additional, Lenardo, Michael J., additional, and Uzel, Gulbu, additional

Published
2023
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48. Contributors

Author

Abers, Michael S., primary, Babushok, Daria V., additional, Ballow, Mark, additional, Boisson, Bertrand, additional, Bonagura, Vincent Robert, additional, Bonilla, Francisco A., additional, Bosco de Oliveira Filho, João, additional, Boztug, Kaan, additional, Broderick, Lori, additional, Butte, Manish J., additional, Candotti, Fabio, additional, Casanova, Jean-Laurent, additional, Chandrakasan, Shanmuganathan, additional, Condino-Neto, Antonio, additional, Crow, Yanick J., additional, Cunningham-Rundles, Charlotte, additional, Dalm, Virgil A.S.H., additional, de Jesus, Adriana A., additional, de Maio, Emma, additional, de Saint Basile, Geneviève, additional, de Vries, Esther, additional, Dokal, Inderjeet, additional, Duncan, Christopher J.A., additional, Durandy, A., additional, Ehl, Stephan, additional, Etzioni, Amos, additional, Ferguson, Polly J., additional, Fleisher, Thomas A., additional, Forbes-Satter, Lisa R., additional, Frank, Michael M., additional, Freeman, Alexandra F., additional, Frémond, Marie-Louise, additional, Frew, John W., additional, Fusaro, Mathieu, additional, Gambineri, Eleonora, additional, Ganetzky, Rebecca D., additional, Gennery, Andrew R., additional, Goldbach-Mansky, Raphaela, additional, Goldstein, Amy C., additional, Graham, John M., additional, Gupton, Stephanie E., additional, Haddad, Elie, additional, Hambleton, Sophie, additional, Hanson, Eric P., additional, Heimall, Jennifer, additional, Helfrich, Miep, additional, Henrickson, Sarah E., additional, Holland, Steven M., additional, Hsu, Amy P., additional, Jyonouchi, Soma, additional, Kashef, Sara, additional, Kelsen, Judith, additional, Khalil, Maya, additional, Klein, Christoph, additional, Kobrynski, Lisa, additional, Kohn, Donald B., additional, Kracker, S., additional, Krueger, James G., additional, Lavoie, Pascal M., additional, Lehman, Heather K., additional, Leiding, Jennifer W., additional, Lenardo, Michael J., additional, Levy, Ofer, additional, Lim, Allison Pecha, additional, Lionakis, Michail S., additional, Lisco, Andrea, additional, Lougaris, Vassilios, additional, Lugo Reyes, Saul O., additional, Markert, M. Louise, additional, Marsh, Rebecca A., additional, McCarthy, Elizabeth A., additional, Meyts, Isabelle, additional, Milito, Cinzia, additional, Milner, Joshua D., additional, Ming, Jeffrey E., additional, Moshous, Despina, additional, Müller, Ludmila, additional, Navrazhina, Kristina, additional, Nichols, Kim E., additional, Notarangelo, Luigi D., additional, Oksenhendler, Eric, additional, Orange, Jordan S., additional, Paganelli, Roberto, additional, Pawelec, Graham, additional, Pentimalli, Tancredi Massimo, additional, Perez, Elena E., additional, Picard, Capucine, additional, Plebani, Alessandro, additional, Porras, Oscar, additional, Przespolewski, Amanda C., additional, Puel, Anne, additional, Pulvirenti, Federica, additional, Quinti, Isabella, additional, Rezaei, Nima, additional, Rijkers, Ger T., additional, Rosenthal, David Walter, additional, Rosenzweig, Sergio D., additional, Segal, Brahm H., additional, Seppänen, Mikko R.J., additional, Sereti, Irini, additional, Shcherbina, Anna, additional, Sobacchi, Cristina, additional, Squire, Jacqueline D., additional, Stepensky, Polina, additional, Su, Helen C., additional, Sullivan, Kathleen E., additional, Torgerson, Troy R., additional, Uzel, Gulbu, additional, van der Burg, Mirjam, additional, Villa, Anna, additional, Villartay, Jean-Pierre de, additional, Warnatz, Klaus, additional, Wasserman, Richard L., additional, Weemaes, Corry M.R., additional, Yu, Joyce E., additional, Zhang, Shen-Ying, additional, and Ziegler, John B., additional

Published
2020
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