Intrathymic delivery a new route for adenoviral-associated vector gene therapy

Severe combined immunodeficiency (SCID) is a heterogeneous group of rare disorders caused by mutations in different genes involved in the development and function of immune cells. Patients with SCID are usually highly susceptible to severe opportunistic infection, and, if untreated, die within 1 year. Since the first successful bone marrow transplants in X-linked SCID and Wiskott-Aldrich syndrome, hematopoietic stem cell (HSC) transplantation...