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.