US EditionDeafness Reversed: Single Injection Brings Hearing Back Within Weeks
- On July 3, 2025, researchers at Karolinska Institutet and Chinese hospitals reported successful gene therapy restoring hearing in ten patients with OTOF-related deafness.
- The treatment employed an engineered adeno-associated virus to introduce a working OTOF gene into the inner ear through a single injection, targeting the genetic mutation responsible for congenital deafness.
- Most patients, aged one to 24, recovered some hearing within a month, with all showing marked improvement after six months and no serious side effects reported.
- The average level of audible sound decreased significantly from a loudness comparable to a motorcycle engine to that of gentle rainfall, and a seven-year-old participant nearly fully regained her hearing, enabling her to have conversations just four months after receiving treatment.
- Researchers plan to monitor the durability of this therapy and are expanding trials to other common deafness genes, reflecting optimism for broader genetic deafness treatments.
14 Articles
14 Articles
Injection restores hearing within a month 'in huge step forward' for patients with genetic deafness
A revolutionary gene therapy has restored hearing in ten patients suffering from congenital deafness, marking a significant breakthrough in treating genetic hearing loss.The groundbreaking treatment, detailed in Nature Medicine, targeted individuals aged between one and 24 years who had severe hearing impairment caused by mutations in the OTOF gene.The therapy involved a single injection of a synthetic virus carrying a functional version of the …
Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results
Gene therapy is a promising approach for hereditary deafness. We recently showed that unilateral AAV1-hOTOF gene therapy with dual adeno-associated virus (AAV) serotype 1 carrying human OTOF transgene is safe and associated with functional improvements in patients with autosomal recessive deafness 9 (DFNB9). The protocol was subsequently amended and approved to allow bilateral gene therapy administration. Here we report an interim analysis of th…
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