St. Jude is trying to find better treatments for children with X-linked severe combined immunodeficiency disease (SCID - Xl). (stjude.org)
In the past 6 years, gene therapy has cured at least 17 children with two types of severe combined immunodeficiency disease (SCID). (sciencemag.org)
1990: The first FDA - approved gene therapy trial takes place at the NIH, when two girls with a type of severe combined immunodeficiency syndrome (SCID) caused by an enzyme deficiency are infused with modified versions of their own blood cells that carries genes for the functioning version of the enzyme. (discovermagazine.com)