P191: Preliminary safety and efficacy of AK-OTOF gene therapy for otoferlin gene (OTOF)-mediated hearing loss

Genetics in Medicine Open 2026 Open access

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Authors: Aaron D. Tward
DOI: 10.1016/j.gimo.2026.103685
Cited by: 0

Abstract

The otoferlin gene (OTOF) encodes otoferlin, a protein critical for signaling at the inner hair cell synapse. Individuals with variants in OTOF typically present with congenital, severe to profound sensorineural hearing loss, with preserved otoacoustic emissions early in life. Recent advances in gene therapy and intracochlear delivery support the potential to restore physiologic hearing in individuals with OTOF-mediated hearing loss using a one-time, local administration of AK-OTOF (AAVAnc80-hOTOF).

Fields of Study & Subjects

Hearing Cochlea Tinnitus Genetics Virus-based gene therapy research RNA Interference and Gene Delivery

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103685–103685
Publication date
1 January 2026
Language
EN