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Constitutive IL-1RA production by modified immune cells protects against IL-1-mediated inflammatory disorders.
Colantuoni M, Jofra Hernandez R, Pettinato E, Basso-Ricci L, Magnani L, Andolfi G, Rigamonti C, Finardi A, Romeo V, Soldi M, Sergi Sergi L, Rocchi M, Scala S, Hoffman HM, Gregori S, Kajaste-Rudnitski A, Sanvito F, Muzio L, Naldini L, Aiuti A, Mortellaro A. Colantuoni M, et al. Among authors: naldini l. Sci Transl Med. 2023 May 31;15(698):eade3856. doi: 10.1126/scitranslmed.ade3856. Epub 2023 May 31. Sci Transl Med. 2023. PMID: 37256935
Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects.
Mortellaro A, Hernandez RJ, Guerrini MM, Carlucci F, Tabucchi A, Ponzoni M, Sanvito F, Doglioni C, Di Serio C, Biasco L, Follenzi A, Naldini L, Bordignon C, Roncarolo MG, Aiuti A. Mortellaro A, et al. Among authors: naldini l. Blood. 2006 Nov 1;108(9):2979-88. doi: 10.1182/blood-2006-05-023507. Epub 2006 Jul 11. Blood. 2006. PMID: 16835374 Free article.
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A. Marangoni F, et al. Among authors: naldini l. Mol Ther. 2009 Jun;17(6):1073-82. doi: 10.1038/mt.2009.31. Epub 2009 Mar 3. Mol Ther. 2009. PMID: 19259069 Free PMC article.
313 results