Lentiviral HSC Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Press brief by Suk See De Ravin, MD, PhD, at the American Society of Hematology (ASH) 2015 annual meeting. Dr De Ravin gives an overview of how lentiviral-gene therapy with reduced-intensity conditioning appears safe and achieves unprecendented pan-immunologic correction of T, B, and NK cell compartments due to engraftment of relatively high levels of transduced HSCs in the bone marrow. This is the first demonstration of the use of gene therapy to salvage failed allogeneic HSCT in older SCID-X1 patients.