‘Bubble boy’ disease erased with simple therapy
New study shows the faulty gene that causes ADA-SCID was successfully replaced in 48 out of 50 patients: NEJM
Dozens of children with so-called ‘bubble boy’ disease have been successfully treated by having the defective gene that causes the condition replaced, new research shows.
A total of 50 children from the US and UK with adenosine deaminase deficiency and severe combined immunodeficiency (ADA-SCID) had the therapy which produces engraftment of cells that corrected the immune system deficiency.
