Search

Your search keyword '"Zancan S"' showing total 7 results
Start Over Author "Zancan S" Database OpenAIRE
7 results on '"Zancan S"'

1. Lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD): Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200

Author

Calbi, V., Francesca Fumagalli, Acquati, S., Miglietta, S., Ciotti, F., Fraschini, M., Sarzana, M., Baldoli, C., Recupero, S., Zambon, A., Barzaghi, F., Ferrua, F., Cicalese, M. P., Migliavacca, M., Tucci, F., Gallo, V., La Marca, G., Silvani, P., Facchini, M., Locatelli, S., Antonioli, G., Zancan, S., Farinelli, G., Morena, F., Segovia, J., Schwab, L. C., Downey, G., Gabaldo, M., Martino, S., Ciceri, F., Sora, M. G. Natali, Bernardo, M. E., Naldini, L., and Aiuti, A.

Published
2019

5. Update on safety and efficacy of lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

Author

Calbi, V., Fumagalli, F., Lorioli, L., maria sessa, Bernardo, M. E., Cugnata, F., Roncoita, P. M. V., Acquati, S., Redaelli, D., Attanasio, V., Penati, R., Cicalese, M. P., Ferrua, F., Barzaghi, F., Migliavacca, M., Tucci, F., Assanelli, A., Silvani, P., Silvani, F., Facchini, M., Zancan, S., Ciotti, F., Sarzana, M., Zambon, A., Calabria, A., Montini, E., Canale, S., Antonioli, G., Gabaldo, M., Lopez, I. D., Morena, F., Quattrini, A., Baldoli, C., Martino, S., Di Serio, C., Ciceri, F., Sora, M. G. Natali, Naldini, L., Biffi, A., and Aiuti, A.

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

Author

Bernhard, Gentner, Francesca, Tucci, Stefania, Galimberti, Francesca, Fumagalli, Maurizio, De Pellegrin, Paolo, Silvani, Chiara, Camesasca, Silvia, Pontesilli, Silvia, Darin, Francesca, Ciotti, Marina, Sarzana, Giulia, Consiglieri, Chiara, Filisetti, Giulia, Forni, Laura, Passerini, Daniela, Tomasoni, Daniela, Cesana, Andrea, Calabria, Giulio, Spinozzi, Maria-Pia, Cicalese, Valeria, Calbi, Maddalena, Migliavacca, Federica, Barzaghi, Francesca, Ferrua, Vera, Gallo, Simona, Miglietta, Erika, Zonari, Patali S, Cheruku, Claudia, Forni, Marcella, Facchini, Ambra, Corti, Michela, Gabaldo, Stefano, Zancan, Serena, Gasperini, Attilio, Rovelli, Jaap-Jan, Boelens, Simon A, Jones, Robert, Wynn, Cristina, Baldoli, Eugenio, Montini, Silvia, Gregori, Fabio, Ciceri, Maria G, Valsecchi, Giancarlo, la Marca, Rossella, Parini, Luigi, Naldini, Alessandro, Aiuti, Maria-Ester, Bernardo, Ilaria, Visagalli, 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, 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. -P., Calbi, V., Migliavacca, M., Barzaghi, F., Ferrua, F., Gallo, V., Miglietta, S., Zonari, E., Cheruku, P. S., Forni, C., Facchini, M., Corti, A., Gabaldo, M., Zancan, S., Gasperini, S., Rovelli, A., Boelens, J. -J., Jones, S. A., Wynn, R., Baldoli, C., Montini, E., Gregori, S., Ciceri, F., Valsecchi, M. G., La Marca, G., Parini, R., Naldini, L., Aiuti, A., and Bernardo, M. -E.

Subjects
Male, Oncology, medicine.medical_specialty, Mucopolysaccharidosis I, Urinary system, Genetic enhancement, Genetic Vectors, Transplantation, Autologous, Iduronidase, Mucopolysaccharidosis type I, Internal medicine, MPSIH, medicine, Humans, Progenitor cell, Hurler syndrome, Glycosaminoglycans, business.industry, Lentivirus, mucopolysaccharidosis type I, Hematopoietic Stem Cell Transplantation, Infant, Genetic Therapy, General Medicine, medicine.disease, gene therapy, Transplantation, Haematopoiesis, Child, Preschool, Mutation, Female, business, Ex vivo, Follow-Up Studies, Stem Cell Transplantation
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 joint stiffness, and normal growth in line with World Health Organization growth charts. Conclusions The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system. (Funded by Fondazione Telethon and others; ClinicalTrials.gov number, ; EudraCT number, .)Hematopoietic Gene Therapy for Hurler Syndrome Eight patients with Hurler syndrome who lacked suitable allogeneic donors received autologous hematopoietic stem and progenitor cells transduced ex vivo with an alpha-L-iduronidase-encoding lentiviral vector. This therapy resulted in extensive metabolic correction in peripheral tissues and the central nervous system.

Published
2021

7. Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

Author

Maddalena Migliavacca, Paola Massariello, Roberto Miniero, Laura Lorioli, Laura Castagnaro, Francesca Tucci, Francesca Fumagalli, Francesco Calzatini, Miriam Casiraghi, Luca Santoleri, Claudia Fossati, Daniele Canarutto, Marcella Facchini, Maria Pia Cicalese, Fabio Ciceri, Alessandra Biffi, Matilde Zambelli, Sarah Marktel, Valeria Calbi, Stefano Zancan, Marta Claudia Frittoli, Paolo Silvani, Gigliola Antonioli, Giulia Consiglieri, Raffaella Milani, Silvia Darin, Francesca Ferrua, Salvatore Recupero, Maria Ester Bernardo, Salvatore Gattillo, Rossana Fiori, Alessandro Aiuti, Federica Barzaghi, Michele Manfredini, Tucci, F., Frittoli, M., Barzaghi, F., Calbi, V., Migliavacca, M., Ferrua, F., Fumagalli, F., Lorioli, L., Castagnaro, L., Facchini, M., Fossati, C., Zancan, S., Massariello, P., Manfredini, M., Consiglieri, G., Canarutto, D., Recupero, S., Calzatini, F., Casiraghi, M., Darin, S., Antonioli, G., Miniero, R., Fiori, R., Silvani, P., Zambelli, M., Marktel, S., Gattillo, S., Milani, R., Santoleri, L., Ciceri, F., Biffi, A., Cicalese, M. P., Bernardo, M. E., and Aiuti, A.

Subjects
Male, medicine.medical_specialty, Transplantation Conditioning, Genetic enhancement, Urology, CD34, Article, 03 medical and health sciences, 0302 clinical medicine, Bone Marrow, Medicine, Humans, Progenitor cell, Transplantation, business.industry, Hematopoietic Stem Cell Transplantation, Hematology, Genetic Therapy, medicine.disease, Metachromatic leukodystrophy, Haematopoiesis, medicine.anatomical_structure, 030220 oncology & carcinogenesis, Female, Bone marrow, Stem cell, business, Ex vivo, 030215 immunology
Abstract

Collection of an adequate amount of autologous haematopoietic stem progenitor cells (HSPC) is required for ex vivo manipulation and successful engraftment for certain inherited disorders. Fifty-seven paediatric patients (age 0.5–11.4 years) underwent a bone marrow harvest for the purpose of HSPC gene therapy (GT), including adenosine deaminase-severe combined immunodeficiency (ADA-SCID), Wiskott–Aldrich syndrome (WAS) and metachromatic leukodystrophy (MLD) patients. Total nucleated cells and the percentage and absolute counts of CD34+ cells were calculated at defined steps of the procedure (harvest, CD34+ cell purification, transduction with the gene transfer vector and infusion of the medicinal product). A minimum CD34+ cell dose for infusion was 2 × 106/kg, with an optimal target at 5–10 × 106/kg. Median volume of bone marrow harvested was 34.2 ml/kg (range 14.2–56.6). The number of CD34+ cells collected correlated inversely with weight and age in all patients and particularly in the MLD children group. All patients reached the minimum target dose for infusion: median dose of CD34+ cells/kg infused was 10.3 × 106/kg (3.7–25.9), with no difference among the three groups. Bone marrow harvest of volumes > 30 ml/kg in infants and children with ADA-SCID, WAS and MLD is well tolerated and allows obtaining an adequate dose of a medicinal product for HSPC-GT.

Published
2019

Catalog

Books, media, physical & digital resources
See catalog results