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Article

Dual AAV-based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F.

Molecular therapy : the journal of the American Society of Gene Therapy - 6 Dec 2023

Riaz Sehar, Sethna Saumil, Duncan Todd, Naeem Muhammad A, Redmond T Michael, Riazuddin Sheikh, Riazuddin Saima, Carvalho Livia S, Ahmed Zubair M

Abstract excerpt

Mutations in the PCDH15 gene, encoding protocadherin-15, are among the leading causes of Usher syndrome type 1 (USH1F), and account for up to 12% USH1 cases worldwide. A founder truncating variant of PCDH15 has a ∼2% carrier frequency in Ashkenazi Jews accounting for nearly 60% of their USH1 cases. Although cochlear implants can restore hearing perception in USH1 patients, presently there are no effective...

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Identifiers

PMID: 37864333
DOI: 10.1016/j.ymthe.2023.10.017
PMCID: PMC10727994

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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