Article

Combination of engineered cell type-specific promoters and a high-efficiency AAV capsid restores hearing in adult DFNB1 mice model with demonstrated safety in nonhuman primate

2026-04-18

Abstract excerpt

A major challenge in gene therapy for GJB2 -related hearing loss (DFNB1)—the most common form of hereditary deafness—is achieving efficient and precise connexin 26 delivery. Herein, we engineered two cell type-specific promoters (GJB2-1 and WFS1-2274) and developed an AAV capsid, AAV-MAS012, with enhanced transduction efficiency in mature cochlear cells. Our AAV-mediated gene therapy systems restored hearing of l...

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Literature Corpus work: 06295f08-1a0d-5d47-bcde-fe7acf612d88
DOI: 10.64898/2026.04.15.718827

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