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Page 1
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A. Brunetti-Pierri N, et al. Among authors: zancan s. NEJM Evid. 2022 Jul;1(7):EVIDoa2200052. doi: 10.1056/EVIDoa2200052. Epub 2022 Jun 6. NEJM Evid. 2022. PMID: 38319253
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: zancan s. N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596. N Engl J Med. 2021. PMID: 34788506 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: zancan s. 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.
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Migliavacca M, Barzaghi F, Fossati C, Rancoita PMV, Gabaldo M, Dionisio F, Giannelli S, Salerio FA, Ferrua F, Tucci F, Calbi V, Gallo V, Recupero S, Consiglieri G, Pajno R, Sambuco M, Priolo A, Ferri C, Garella V, Monti I, Silvani P, Darin S, Casiraghi M, Corti A, Zancan S, Levi M, Cesana D, Carlucci F, Pituch-Noworolska A, AbdElaziz D, Baumann U, Finocchi A, Cancrini C, Ladogana S, Meinhardt A, Meyts I, Montin D, Notarangelo LD, Porta F, Pasquet M, Speckmann C, Stepensky P, Tommasini A, Rabusin M, Karakas Z, Galicchio M, Leonardi L, Duse M, Guner SN, Di Serio C, Ciceri F, Bernardo ME, Aiuti A, Cicalese MP. Migliavacca M, et al. Among authors: zancan s. Nat Med. 2024 Feb;30(2):488-497. doi: 10.1038/s41591-023-02789-4. Epub 2024 Feb 14. Nat Med. 2024. PMID: 38355973
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: zancan s. 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.
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: zancan s. 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.
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.
Stirnadel-Farrant H, Kudari M, Garman N, Imrie J, Chopra B, Giannelli S, Gabaldo M, Corti A, Zancan S, Aiuti A, Cicalese MP, Batta R, Appleby J, Davinelli M, Ng P. Stirnadel-Farrant H, et al. Among authors: zancan s. Orphanet J Rare Dis. 2018 Apr 6;13(1):49. doi: 10.1186/s13023-018-0791-9. Orphanet J Rare Dis. 2018. PMID: 29625577 Free PMC article.
Safety Profile and Immunologic Responses of a Novel Vaccine Against Shigella sonnei Administered Intramuscularly, Intradermally and Intranasally: Results From Two Parallel Randomized Phase 1 Clinical Studies in Healthy Adult Volunteers in Europe.
Launay O, Lewis DJM, Anemona A, Loulergue P, Leahy J, Sciré AS, Maugard A, Marchetti E, Zancan S, Huo Z, Rondini S, Marhaba R, Finco O, Martin LB, Auerbach J, Cohen D, Saul A, Gerke C, Podda A. Launay O, et al. Among authors: zancan s. EBioMedicine. 2017 Aug;22:164-172. doi: 10.1016/j.ebiom.2017.07.013. Epub 2017 Jul 15. EBioMedicine. 2017. PMID: 28735965 Free PMC article. Clinical Trial.
Functional effects of chronic paroxetine versus placebo on the fear, stress and anxiety brain circuit in Social Anxiety Disorder: initial validation of an imaging protocol for drug discovery.
Giménez M, Ortiz H, Soriano-Mas C, López-Solà M, Farré M, Deus J, Martín-Santos R, Fernandes S, Fina P, Bani M, Zancan S, Pujol J, Merlo-Pich E. Giménez M, et al. Among authors: zancan s. Eur Neuropsychopharmacol. 2014 Jan;24(1):105-16. doi: 10.1016/j.euroneuro.2013.09.004. Epub 2013 Oct 14. Eur Neuropsychopharmacol. 2014. PMID: 24332890 Clinical Trial.
11 results