'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.
David Vetter, who had SCID, died aged 12 in 1984.
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