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Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
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Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years

Nature·Shu, Yilai·6 months ago
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Data availability

Participant data are de-identified and available in the text, figures and tables. The statistical analysis plan is included in the clinical trial protocol, which is available in Supplementary Information . Requests for more information should be communicated to the corresponding author Y.S. Source data are provided with this paper.

Code availability

This work does not include any new code or software.

References

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).

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Lv, J. et al. AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. Lancet 403 , 2317–2325 (2024).

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Wang, H. et al. Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results. Nat. Med. 30 , 1898–1904 (2024).

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Cheng, X. et al.…

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