Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Abstract

Treatment of ADA-SCID with ex vivo lentiviral HSPC gene therapy resulted in high overall and event-free survival with sustained <i>ADA</i> expression, metabolic correction, and functional immune reconstitution. (Funded by the National Institutes of Health and others; ClinicalTrials.gov numbers, NCT01852071, NCT02999984, and NCT01380990.).