"Gene Therapy Restores Hearing in Children with Hereditary Deafness"

An 11-year-old boy in the US, born profoundly deaf due to a rare gene abnormality, can now hear after receiving a groundbreaking gene therapy at the Children's Hospital of Philadelphia. The therapy involved injecting a modified virus carrying a working copy of the otoferlin gene into his cochlea, enabling his inner ear hair cells to produce the missing protein and function properly. While his hearing has significantly improved, it's uncertain if he will learn to speak due to the late intervention. This milestone offers hope for patients worldwide with genetic hearing loss, and similar trials are underway in the US, Europe, and China.