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102 results

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Page 1
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
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 MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Miglietta S, Zonari E, Cheruku PS, Forni C, Facchini M, Corti A, Gabaldo M, Zancan S, Gasperini S, Rovelli A, Boelens JJ, Jones SA, Wynn R, Baldoli C, Montini E, Gregori S, Ciceri F, Valsecchi MG, la Marca G, Parini R, Naldini L, Aiuti A, Bernardo ME; MPSI Study Group. Gentner B, et al. Among authors: cicalese mp. N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596. N Engl J Med. 2021. PMID: 34788506 Clinical Trial.
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L. Biffi A, et al. Among authors: cicalese mp. Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11. Science. 2013. PMID: 23845948 Clinical Trial.
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.
Quaranta P, Basso-Ricci L, Jofra Hernandez R, Pacini G, Naldini MM, Barcella M, Seffin L, Pais G, Spinozzi G, Benedicenti F, Pietrasanta C, Cheong JG, Ronchi A, Pugni L, Dionisio F, Monti I, Giannelli S, Darin S, Fraschetta F, Barera G, Ferrua F, Calbi V, Ometti M, Di Micco R, Mosca F, Josefowicz SZ, Montini E, Calabria A, Bernardo ME, Cicalese MP, Gentner B, Merelli I, Aiuti A, Scala S. Quaranta P, et al. Among authors: cicalese mp. Blood. 2024 May 9;143(19):1937-1952. doi: 10.1182/blood.2023022666. Blood. 2024. PMID: 38446574 Free PMC article. Clinical Trial.
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Cesana D, Cicalese MP, Calabria A, Merli P, Caruso R, Volpin M, Rudilosso L, Migliavacca M, Barzaghi F, Fossati C, Gazzo F, Pizzi S, Ciolfi A, Bruselles A, Tucci F, Spinozzi G, Pais G, Benedicenti F, Barcella M, Merelli I, Gallina P, Giannelli S, Dionisio F, Scala S, Casiraghi M, Strocchio L, Vinti L, Pacillo L, Draghi E, Cesana M, Riccardo S, Colantuono C, Six E, Cavazzana M, Carlucci F, Schmidt M, Cancrini C, Ciceri F, Vago L, Cacchiarelli D, Gentner B, Naldini L, Tartaglia M, Montini E, Locatelli F, Aiuti A. Cesana D, et al. Among authors: cicalese mp. Nat Commun. 2024 Apr 30;15(1):3662. doi: 10.1038/s41467-024-47866-5. Nat Commun. 2024. PMID: 38688902 Free PMC article.
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: cicalese mp. 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.
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
Fumagalli F, Calbi V, Gallo V, Zambon AA, Recupero S, Ciotti F, Sarzana M, Fraschini M, Scarparo S, De Mattia F, Miglietta S, Pierini C, Soncini M, Morena F, Montini E, Barzaghi F, Consiglieri G, Ferrua F, Migliavacca M, Tucci F, Fratini ES, Ippolito A, Silvani P, Calvi MR, Clerici A, Corti A, Facchini M, Locatelli S, Sangalli M, Zancan S, Miotto F, Natali Sora MG, Baldoli C, Martino S, Córdoba-Claros A, Moro SL, Gollop ND, Abate J, Yarzi MN, Nutkins P, Shenker A, Calissano M, Brooks J, Richardson A, Campbell L, Filippi M, Naldini L, Cicalese MP, Ciceri F, Bernardo ME, Aiuti A. Fumagalli F, et al. Among authors: cicalese mp. N Engl J Med. 2025 Apr 24;392(16):1609-1620. doi: 10.1056/NEJMoa2405727. N Engl J Med. 2025. PMID: 40267426 Clinical Trial.
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME. Consiglieri G, et al. Among authors: cicalese mp. Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1. Sci Transl Med. 2024. PMID: 38691622 Clinical Trial.
Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series.
Naviglio S, Cicalese MP, Rivers E, Ferrua F, Bonfim C, Cenciarelli S, Cheong KN, Faraci M, Giardino S, Ghosh S, Lee PP, Lyra PT, Meisel R, Sofia V, Tessitore A, Tommasini A, Valencic E, Vallée TC, Volpi S, Worth AJ, Rabusin M, Albert MH, Thrasher AJ, Aiuti A. Naviglio S, et al. Among authors: cicalese mp. Blood. 2024 Oct 17;144(16):1699-1704. doi: 10.1182/blood.2024024524. Blood. 2024. PMID: 39046813
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity.
Bognàr T, Garcia-Rosa M, Lalmohamed A, Güngör T, Hauri-Hohl M, Prockop S, Oram L, Pai SY, Brooks J, Savic RM, Dvorak CC, Long-Boyle JR, Krajinovic M, Bittencourt H, Teyssier AC, Théorêt Y, Martinez C, Egberts TCG, Morales E, Slatter M, Cuvelier GDE, Chiesa R, Wynn RF, Coussons M, Cicalese MP, Ansari M, Long SE, Ebens CL, Lust H, Chaudhury S, Nath CE, Shaw PJ, Keogh SJ, van der Stoep MYEC, Bredius R, Lindemans CA, Boelens JJ, Bartelink IH. Bognàr T, et al. Among authors: cicalese mp. Blood Adv. 2024 Oct 8;8(19):5137-5145. doi: 10.1182/bloodadvances.2024013275. Blood Adv. 2024. PMID: 39074263 Free PMC article.
102 results