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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: farinelli 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.
A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.
Migliavacca M, Basso Ricci L, Farinelli G, Calbi V, Tucci F, Barzaghi F, Ferrua F, Cicalese MP, Darin S, Barzaghi LR, Giglio F, Peccatori J, Fumagalli F, Nicoletti R, Giannelli S, Sartirana C, Bandiera A, Esposito M, Milani R, Mazzi B, Finocchi A, Marktel S, Assanelli A, Locatelli F, Ciceri F, Aiuti A, Bernardo ME. Migliavacca M, et al. Among authors: farinelli g. J Clin Immunol. 2022 Nov;42(8):1742-1747. doi: 10.1007/s10875-022-01338-x. Epub 2022 Aug 10. J Clin Immunol. 2022. PMID: 35945378 Free PMC article.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Jofra Hernández R, Calabria A, Sanvito F, De Mattia F, Farinelli G, Scala S, Visigalli I, Carriglio N, De Simone M, Vezzoli M, Cecere F, Migliavacca M, Basso-Ricci L, Omrani M, Benedicenti F, Norata R, Rancoita PMV, Di Serio C, Albertini P, Cristofori P, Naldini L, Gentner B, Montini E, Aiuti A, Mortellaro A. Jofra Hernández R, et al. Among authors: farinelli g. Mol Ther. 2021 Jan 6;29(1):86-102. doi: 10.1016/j.ymthe.2020.09.030. Epub 2020 Sep 23. Mol Ther. 2021. PMID: 33010230 Free PMC article.
23 results