Gene therapy offers cure for an immune disorder
Genetically modified stem cells restore immune function in more than 95% of patients with the rare disease.
Dr. Donald Kohn with a member of his lab. Credit: Ann Johansson/UCLA Broad Stem Cell Research Center
An international team of scientists have developed an experimental gene therapy treatment for children with a severe immune disorder, and it’s showing promise.
As described in a paper published in the
New England Journal of Medicine, clinical trials in the US and UK have successfully treated 48 out of 50 participants born with a rare and life-threatening disease known as severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).