Deafness reversed: One injection restores hearing in just weeks
Why it matters: This gene therapy offers a life-changing solution for thousands born with OTOF-related deafness.
- Karolinska Institutet researchers, in collaboration with Chinese hospitals, conducted a study where a single injection of gene therapy restored hearing in individuals born deaf due to OTOF gene mutations.
- Ten patients, ranging from 1 to 24 years old, received the treatment, with all showing improved hearing within one month and significant gains after six months, averaging an improvement from 106 to 52 decibels.
- Younger patients, particularly those aged five to eight, demonstrated the most dramatic responses, with one seven-year-old regaining nearly full hearing, though adults also experienced meaningful improvements.
- The therapy, which delivers a working copy of the OTOF gene using a synthetic adeno-associated virus (AAV) directly into the inner ear, was found to be safe and well-tolerated.
A groundbreaking gene therapy, delivered via a single injection, has reversed deafness in patients with OTOF gene mutations, with all ten participants experiencing significant hearing improvement within weeks. This study, led by Karolinska Institutet and collaborators in China, marks a major advancement in genetic deafness treatment for both children and adults.
