Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

Gene therapy

Gene therapy for hereditary deafness

Bilateral administration of human OTOF gene therapy appears safe, and preliminary data suggest that it could effectively treat a rare form of hereditary hearing loss by enhancing speech perception, sound source localization and overall auditory experience.

This is a preview of subscription content, access via your institution

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

Fig. 1: Gene therapy via AAV injection into the inner ear.

References

  1. Petit, C., Bonnet, C. & Safieddine, S. Nat. Rev. Genet. 24, 665–686 (2023).

    Article  CAS  PubMed  Google Scholar 

  2. Yasunaga, S. et al. Nat. Genet. 21, 363–369 (1999).

    Article  CAS  PubMed  Google Scholar 

  3. Pangršič, T. et al. Nat. Neurosci. 13, 869–876 (2010).

    Article  PubMed  Google Scholar 

  4. Ford, C. L., Riggs, W. J., Quigley, T., Keifer, O. P., Whitton, J. P. & Valayannopoulos, V. Hum. Genet. 142, 1429–1449 (2023).

    Article  PubMed  PubMed Central  Google Scholar 

  5. Ramzan, M. et al. J. Hum. Genet. 68, 657–669 (2023).

    Article  CAS  PubMed  Google Scholar 

  6. Wang, H. C. et al. Nat. Med. https://doi.org/10.1038/s41591-024-03023-5 (2024).

  7. Hahn, R. & Avraham, K. B. Audiol. Res. 13, 952–966 (2023).

    Article  PubMed  PubMed Central  Google Scholar 

  8. Akil, O. et al. Proc. Natl Acad. Sci. USA 116, 4496–4501 (2019).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  9. Al‐Moyed, H., Cepeda, A. P., Jung, S., Moser, T., Kügler, S. & Reisinger, E. EMBO Mol. Med. 11, e9396 (2019).

    Article  PubMed  Google Scholar 

  10. Lv, J. et al. Lancet https://doi.org/10.1016/S0140-6736(23)02874-X (2024).

    Article  PubMed  Google Scholar 

  11. Qi, J. et al. Adv. Sci. 11, e2306788 (2024).

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Mustafa Tekin.

Ethics declarations

Competing interests

The authors declare no competing interests.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Smith, C., Zafeer, M.F. & Tekin, M. Gene therapy for hereditary deafness. Nat Med (2024). https://doi.org/10.1038/s41591-024-03004-8

Download citation

  • Published:

  • DOI: https://doi.org/10.1038/s41591-024-03004-8

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing