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A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB.
Crippa S, Alberti G, Passerini L, Savoia EO, Mancino M, De Ponti G, Santi L, Berti M, Testa M, Hernandez RJ, Quaranta P, Ceriotti S, Visigalli I, Morrone A, Paoli A, Forni C, Scala S, Degano M, Staiano L, Gregori S, Aiuti A, Bernardo ME. Crippa S, et al. Among authors: bernardo me. Mol Ther Methods Clin Dev. 2024 Aug 6;32(3):101313. doi: 10.1016/j.omtm.2024.101313. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39282079 Free PMC article.
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: bernardo me. Blood. 2024 May 9;143(19):1937-1952. doi: 10.1182/blood.2023022666. Blood. 2024. PMID: 38446574 Free PMC article. 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: bernardo me. 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.
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.
Canarutto D, Oltolini C, Barzaghi F, Calbi V, Migliavacca M, Tucci F, Gallo V, Consiglieri G, Ferrua F, Recupero S, Cervi MC, Al-Mousa H, Pituch-Noworolska A, Tassan Din C, Scarpellini P, Silvani P, Fossati C, Casiraghi M, Cirillo DM, Castagna A, Bernardo ME, Aiuti A, Cicalese MP. Canarutto D, et al. Among authors: bernardo me. Biomedicines. 2023 Jun 24;11(7):1809. doi: 10.3390/biomedicines11071809. Biomedicines. 2023. PMID: 37509449 Free PMC article.
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. Among authors: bernardo me. Front Immunol. 2023 Jun 26;14:1187959. doi: 10.3389/fimmu.2023.1187959. eCollection 2023. Front Immunol. 2023. PMID: 37435083 Free PMC article. Review.
Current practices for nutritional evaluation and care during the treatment of pediatric oncology patients: a survey among AIEOP centers.
Venturelli F, Leardini D, Baccelli F, Gottardi F, Barat V, Vendemini F, Folsi VM, Meazza C, Marinoni M, Bernardo ME, Tumino M, Brugiolo A, Pizzato C, Battisti L, Bertolini P, Pancaldi A, Rinieri S, Pericoli R, Coccia P, Onofrillo D, Fabozzi F, Bianchi S, Rizzo D, Daniele RM, Stellato P, Prete A, Masetti R, Muratore E. Venturelli F, et al. Among authors: bernardo me. Eur J Pediatr. 2024 Mar;183(3):1137-1144. doi: 10.1007/s00431-023-05368-4. Epub 2023 Dec 14. Eur J Pediatr. 2024. PMID: 38095717
Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities.
Bulté D, Barzaghi F, Mesa-Nuñez C, Rigamonti C, Basso-Ricci L, Visconti C, Crippa S, Pettinato E, Gilioli D, Milani R, Quaranta P, Caorsi R, Cafaro A, Cangemi G, Lupia M, Schena F, Grossi A, Di Colo G, Federici S, Insalaco A, De Benedetti F, Marktel S, Di Micco R, Bernardo ME, Scala S, Cicalese MP, Conti F, Miano M, Gattorno M, Dufour C, Aiuti A, Mortellaro A. Bulté D, et al. Among authors: bernardo me. J Allergy Clin Immunol. 2024 Sep 14:S0091-6749(24)00945-X. doi: 10.1016/j.jaci.2024.09.007. Online ahead of print. J Allergy Clin Immunol. 2024. PMID: 39284370 Free article.
136 results