AAVAnc80-hOTOF is an experimental gene therapy for otoferlin gene-mediated hearing loss.[1][2]

References

  1. ↑ Jiang, Luoying; Wang, Daqi; He, Yingzi; Shu, Yilai (April 2023). "Advances in gene therapy hold promise for treating hereditary hearing loss". Molecular Therapy. 31 (4): 934–950. doi:10.1016/j.ymthe.2023.02.001. PMC 10124073. PMID 36755494.
  2. ↑ Hickox, Ann E.; Valero, Michelle D.; McLaughlin, James T.; Robinson, Gregory S.; Wellman, Jennifer A.; McKenna, Michael J.; Sewell, William F.; Simons, Emmanuel J. (November 2021). "Genetic Medicine for Hearing Loss: OTOF as Exemplar". Journal of the American Academy of Audiology. 32 (10): 646–653. doi:10.1055/s-0041-1730410.
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