95% of kids with "bubble boy" disease cured by one-time gene therapy
a day ago
- #gene therapy
- #ADA-SCID
- #medical breakthrough
- A one-time gene therapy using a patient’s own stem cells has effectively cured ADA-SCID in 95% of treated children.
- ADA-SCID is a severe immune disorder, often fatal in early childhood without treatment.
- Current treatments include lifelong enzyme replacement therapy or risky bone marrow transplants requiring matched donors.
- The new gene therapy involves modifying the patient’s own stem cells to produce the missing ADA enzyme.
- 62 children participated in the trial, with a 95% success rate and no need for further treatments.
- Patients showed normal immune function, responded to vaccines, and had no cancer-like cell growth.
- Cryopreservation of stem cells allows broader access to the therapy by enabling local collection and remote processing.
- The therapy is now seeking FDA approval, with hopes for commercialization within 2-3 years.
- The study was published in the New England Journal of Medicine and funded by multiple health organizations.