Gene Therapy for X-SCID
June 4, 2019 | Terry Sharrer
David Vetter (1971-1984), aka “the Boy in the Bubble,” lived with and died from x-linked Severe Combined Immunodeficiency, which has since been a target for gene therapy treatment. This past April, research physicians at UCSF and St. Jude’s Children’s Hospital announced their success in a trial of ten afflicted infants. Key to their strategy was using the alkylating agent busulfan to knock down immune cell precursors, and then introducing a recombinant lentivirus vector which restored function to T cells, B cells, and natural killer cells. All patients showed rising immunological competency. MORE
Image Credit: Flikr
Key words: