Tag: Gene Therapy

Groundbreaking Diabetes Cure: First Patient Produces Own Insulin After Gene-Edited Cell Transplant

A man with type 1 diabetes becomes the first to produce his own insulin after receiving gene-edited islet cell transplants — without immune-suppressing drugs. The breakthrough, published in the New England Journal of Medicine, could revolutionize diabetes treatment worldwide.

DB-OTO Gene Therapy Restores Hearing in Children with Genetic Deafness – NEJM Study 2025

A new NEJM study shows that DB-OTO, a dual AAV1 gene therapy, restored natural hearing in 75% of children with otoferlin-related inherited deafness. This breakthrough could transform treatment for congenital hearing loss.