articleNature MedicineJun 5, 2024HYBRID OA

Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results

HWHui WangYCYuxin ChenJLJun LvXCXiaoting ChengQCQi Cao

Fudan University · Eye & ENT Hospital of Fudan University · +6 more institutions

PubMed
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Abstract

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 the single-arm trial investigating the safety and efficacy of binaural therapy in five pediatric patients with DFNB9. The primary endpoint was dose-limiting toxicity at 6 weeks, and the secondary endpoint included safety (adverse events) and efficacy (auditory function…

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Topics & keywords

Topics
Keywords
  • Adverse effect
  • Medicine
  • Clinical endpoint
  • Auditory brainstem response
  • Audiology
  • Interim analysis
  • Genetic enhancement
  • Hearing loss
UN Sustainable Development Goals
  • Good health and well-being
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