Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects.
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C. Di Serio | L. Biasco | L. Naldini | C. Doglioni | A. Mortellaro | M. Roncarolo | C. Bordignon | F. Sanvito | A. Aiuti | M. Ponzoni | R. J. Hernández | A. Follenzi | M. Guerrini | F. Carlucci | A. Tabucchi