Work

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

Year: 2026

Type: article

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.

Source: Signal Transduction and Targeted Therapy

DOI: 10.1038/s41392-026-02942-8

Authors: Bikash R. Pattnaik, Jose A. Sahel

Institutions: University of Wisconsin–Madison, University of Pittsburgh

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,790