Article

Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and non-human primates

2023-08-23

Abstract excerpt

Pathogenic mutations in the OTOF gene cause autosomal recessive hearing loss 9 (DFNB9), one of the most common forms of auditory neuropathy. There is no biological treatment for DFNB9. Here, we designed an OTOF gene therapy agent by dual AAV1 carrying human OTOF coding sequences with the expression driven by the hair cell-specific promoter Myo15 , AAV1-hOTOF. To develop a clinical application of AAV1-hOTOF gen...

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Literature Corpus work: 00efe186-21b6-5f44-b58d-a6e50d989921
DOI: 10.1101/2023.08.22.554252

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