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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: fratini es. 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.
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.
Fratini ES, Migliavacca M, Barzaghi F, Fossati C, Giannelli S, Monti I, Casiraghi M, Ferrua F, Recupero S, Consiglieri G, Calbi V, Tucci F, Gallo V, Bernardo ME, Cenciarelli S, Palmoni M, Moni M, Galimberti L, Duse M, Leonardi L, Sieni E, Soncini E, Porta F, Notarangelo LD, De Santis R, Ladogana S, Aiuti A, Cicalese MP. Fratini ES, et al. Front Immunol. 2023 Jun 26;14:1187959. doi: 10.3389/fimmu.2023.1187959. eCollection 2023. Front Immunol. 2023. PMID: 37435083 Free PMC article. Review.
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report.
Cenciarelli S, Calbi V, Barzaghi F, Bernardo ME, Oltolini C, Migliavacca M, Gallo V, Tucci F, Fraschetta F, Albertazzi E, Fratini ES, Consiglieri G, Giannelli S, Dionisio F, Sartirana C, Racca S, Camesasca C, Peretto G, Daverio R, Esposito A, De Cobelli F, Silvani P, Rabusin M, Cara A, Trabattoni D, Dispinseri S, Scarlatti G, Piemonti L, Lampasona V, Cicalese MP, Aiuti A, Ferrua F. Cenciarelli S, et al. Among authors: fratini es. Front Immunol. 2020 Nov 24;11:603428. doi: 10.3389/fimmu.2020.603428. eCollection 2020. Front Immunol. 2020. PMID: 33329599 Free PMC article.
Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.
Tucci F, Gallo V, Barzaghi F, Ferrua F, Migliavacca M, Calbi V, Doglio M, Fratini ES, Karakas Z, Guner S, Zambelli M, Parisi C, Milani R, Gattillo S, Mazzi B, Oltolini C, Barbera M, Baldoli C, Cirillo DM, Asnaghi V, De Min C, Cicalese MP, Ciceri F, Aiuti A, Bernardo ME. Tucci F, et al. Among authors: fratini es. Haematologica. 2021 Feb 1;106(2):641-646. doi: 10.3324/haematol.2020.255620. Haematologica. 2021. PMID: 32817285 Free PMC article.