Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results
Hui Wang,
Yuxin Chen,
Jun Lv
et al.
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 binaur… Show more
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