Gene therapy cures 95% of children with rare immune condition.
Gene therapy shows 100% survival and 95% cure rate in children with ADA-SCID over 7.5 years.
Why it matters
- First large-scale study showing long-term safety and efficacy of gene therapy for ADA-SCID.
- Offers a potential cure for a previously life-threatening condition.
- Demonstrates promise for gene therapy in treating other genetic diseases.
By the numbers
- 62 patients treated.
- 7.5 years average follow-up.
- 100% survival rate.
- 95% cure rate.
The big picture
- ADA-SCID is a rare, life-threatening immune deficiency.
- Gene therapy could replace current treatments like enzyme replacement and bone marrow transplants.
- Efforts are underway to make this therapy more widely available.
What they're saying
- Experts highlight the potential of gene therapy for other diseases, including cancers.
- Ethical considerations and accessibility remain important topics.
Caveats
- Study focuses on a specific rare condition.
- Long-term effects are still being studied.
What’s next
- Plans to seek market authorization in the UK.
- Continued research into gene therapy for other conditions.