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Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli F, Calbi V, Natali Sora MG, Sessa M, Baldoli C, Rancoita PMV, Ciotti F, Sarzana M, Fraschini M, Zambon AA, Acquati S, Redaelli D, Attanasio V, Miglietta S, De Mattia F, Barzaghi F, Ferrua F, Migliavacca M, Tucci F, Gallo V, Del Carro U, Canale S, Spiga I, Lorioli L, Recupero S, Fratini ES, Morena F, Silvani P, Calvi MR, Facchini M, Locatelli S, Corti A, Zancan S, Antonioli G, Farinelli G, Gabaldo M, Garcia-Segovia J, Schwab LC, Downey GF, Filippi M, Cicalese MP, Martino S, Di Serio C, Ciceri F, Bernardo ME, Naldini L, Biffi A, Aiuti A. Fumagalli F, et al. Among authors: antonioli g. Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1. Lancet. 2022. PMID: 35065785 Free PMC article. Clinical Trial.
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A. Sessa M, et al. Among authors: antonioli g. Lancet. 2016 Jul 30;388(10043):476-87. doi: 10.1016/S0140-6736(16)30374-9. Epub 2016 Jun 8. Lancet. 2016. PMID: 27289174 Clinical Trial.
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME. Calbi V, et al. Among authors: antonioli g. Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. Bone Marrow Transplant. 2018. PMID: 29379168 Free PMC article. No abstract available.
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
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 MP, Bernardo ME, Aiuti A. Tucci F, et al. Among authors: antonioli g. Bone Marrow Transplant. 2019 Dec;54(12):1995-2003. doi: 10.1038/s41409-019-0573-6. Epub 2019 May 31. Bone Marrow Transplant. 2019. PMID: 31150018 Free PMC article.
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
Fumagalli F, Zambon AA, Rancoita PMV, Baldoli C, Canale S, Spiga I, Medaglini S, Penati R, Facchini M, Ciotti F, Sarzana M, Lorioli L, Cesani M, Natali Sora MG, Del Carro U, Cugnata F, Antonioli G, Recupero S, Calbi V, Di Serio C, Aiuti A, Biffi A, Sessa M. Fumagalli F, et al. Among authors: antonioli g. J Inherit Metab Dis. 2021 Sep;44(5):1151-1164. doi: 10.1002/jimd.12388. Epub 2021 May 4. J Inherit Metab Dis. 2021. PMID: 33855715
102 results