There are now five treatments approved and currently available for commercialization, i.e., Luxturna, Zolgensma, the two chimeric antigen receptor T cell (CAR-T) therapies (Yescarta and Kymriah), and Strimvelis (the gammaretrovirus approved for adenosine deaminase-severe combined immunodeficiency (ADA-SCID) in Europe). The gene discussed is ADA; the disease is severe combined immunodeficiency.