From China: Bilateral Gene Therapy Safely Restores Hearing in Children Born Deaf

For pediatric patients with autosomal recessive deafness 9 (DFNB9), bilateral gene therapy with adeno-associated virus (AAV) serotype 1 carrying human OTOF transgene is safe and efficacious, according to a study published online June 5 in Nature Medicine.

Hui Wang, M.D., from the Eye & ENT Hospital of Fudan University in Shanghai, and colleagues report on the results of an interim analysis of a single-arm trial examining the safety and efficacy of binaural therapy among five pediatric patients with DFNB9.

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