Reprogramming translation for rare disease therapy: challenges posed by large genes

A recent study published in Nature by Jiang et al. delineates a breakthrough in the treatment of inherited hearing loss through gene therapy. 1 This study offers the most compelling clinical evidence so far that gene replacement therapy can safely restore significant hearing in children with OTOF-related deafness, with benefits lasting up to 2.5 years, setting a new standard for treating inherited sensory disorders.

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Publication Details

Journal
Signal Transduction and Targeted Therapy
Published
2026-08-26
DOI
https://doi.org/10.1038/s41392-026-02942-8
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.00

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article

Reprogramming translation for rare disease therapy: challenges posed by large genes

José‐Alain Sahel, Bikash R. Pattnaik
Signal Transduction and Targeted Therapy
CRISPR and Genetic Engineering
article

Reprogramming translation for rare disease therapy: challenges posed by large genes

José‐Alain Sahel, Bikash R. Pattnaik
article en

Abstract

A recent study published in Nature by Jiang et al. delineates a breakthrough in the treatment of inherited hearing loss through gene therapy. 1 This study offers the most compelling clinical evidence so far that gene replacement therapy can safely restore significant hearing in children with OTOF-related deafness, with benefits lasting up to 2.5 years, setting a new standard for treating inherited sensory disorders.

Signal Transduction and Targeted TherapyVol. 11(1)
University of Wisconsin–Madison (US), University of Pittsburgh (US), UPMC Health System (US), University of Pittsburgh Medical Center (US), Smith-Kettlewell Eye Research Institute (US)
Eye and Ear Foundation of Pittsburgh, Research to Prevent Blindness, University of Pittsburgh, Retina Research Foundation
Openalex Percentile: Top 17%
CRISPR and Genetic Engineering
DOIOpen Access PDFOpenAlex
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