‘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.
The condition was erased in 48 of them, reveal results released at the virtual annual meeting of the American Society of Gene and Cell Therapy and published in the New England Journal of Medicine.
To call it a cure “implies lifelong permanence. We don’t know that” cautioned chief author Dr Donald Kohn of the University of California, Los Angeles.
