Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
2 institutional releases
Published in New England Journal of Medicine · View the paper (DOI)
A child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy, showcasing the power of tailored gene editing to treat patients. The infant is now growing well and thriving after receiving three doses of the therapy with no serious side effects.
Coverage from 2 institutions
- World's first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia Children's Hospital of Philadelphia · May 15, 2025 · first to report
- Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment NIH/Office of the Director · May 15, 2025