Congenital deficiency of the purine metabolic enzyme ADA causes severe combined immunodeficiency (SCID). The profound T lymphopenia characteristic of this disease has limited direct investigation ...

Results from a more recent study [9] show that transduction of hematopoietic precursors in 2 patients with severe ADA-deficient SCID was successful and led to engraftment of ADA-producing cells ...

or SCID, called ADA-SCID. All 50 kids were still alive at least two years after treatment, and over 95% required no additional treatment. Despite the success, a tiny biotech company gave up on ...

South San Francisco, CA, November 21, 2024 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has approved ...