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Page 1
Gene therapy for adenosine deaminase deficiency.
Aiuti A, Ficara F, Cattaneo F, Bordignon C, Roncarolo MG. Aiuti A, et al. Curr Opin Allergy Clin Immunol. 2003 Dec;3(6):461-6. doi: 10.1097/00130832-200312000-00007. Curr Opin Allergy Clin Immunol. 2003. PMID: 14612670 Review.
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: aiuti a. 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.
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.
Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C. Aiuti A, et al. J Clin Invest. 2007 Aug;117(8):2233-40. doi: 10.1172/JCI31666. J Clin Invest. 2007. PMID: 17671653 Free PMC article. Clinical Trial.
305 results