Challenges in the development of treatments for hereditary hearing loss include the exploration of the underlying pathological mechanisms, the comprehensive evaluation of safety and efficacy of gene therapies in clinical trials, the optimization of surgical approaches for drug delivery, and effective collaboration between industry and academia.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$32.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on SpringerLink
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Lv, J. et al. AAV1–hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. Lancet 403, 2317–2325 (2024).
Wang, H. et al. Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results. Nat. Med. 30, 1898–1904 (2024).
Qi, J. et al. AAV-mediated gene therapy restores hearing in patients with DFNB9 deafness. Adv. Sci. 11, e2306788 (2024).
Gene therapy restores hearing. Nat. Biotechnol. 42, 829 (2024).
Petit, C., Bonnet, C. & Safieddine, S. Deafness: from genetic architecture to gene therapy. Nat. Rev. Genet. 24, 665–686 (2023).
Yasunaga, S. et al. A mutation in OTOF, encoding otoferlin, a FER-1-like protein, causes DFNB9, a nonsyndromic form of deafness. Nat. Genet. 21, 363–369 (1999).
Al-Moyed, H. et al. A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice. EMBO Mol. Med. 11, e9396 (2019).
Akil, O. et al. Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model. Proc. Natl Acad. Sci. USA 116, 4496–4501 (2019).
Tang, H. et al. Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin. Hum. Genet. 142, 289–304 (2023).
Gao, X. et al. Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents. Nature 553, 217–221 (2018).
Doxzen, K. W. et al. The translational gap for gene therapies in low- and middle-income countries. Sci. Transl. Med. 16, eadn1902 (2024).
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
The authors declare no competing interests.
Rights and permissions
About this article
Cite this article
Chen, Y., Zhong, J. & Shu, Y. Gene therapy for deafness: we can do more. Nat Rev Genet 26, 225–226 (2025). https://doi.org/10.1038/s41576-024-00809-8
Published:
Issue date:
DOI: https://doi.org/10.1038/s41576-024-00809-8
