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Article

Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B.

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

Schott Juliane W, Huang Peixin, Morgan Michael, Nelson-Brantley Jennifer, Koehler Ally, Renslo Bryan, Büning Hildegard, Warnecke Athanasia, Schambach Axel, Staecker Hinrich

Abstract excerpt

Usher syndrome 1B (USH1B) is a devastating genetic disorder with congenital deafness, loss of balance, and blindness caused by mutations in the myosin-VIIa (MYO7A) gene, for which there is currently no cure. We developed a gene therapy approach addressing the vestibulo-cochlear deficits of USH1B using a third-generation, high-capacity lentiviral vector system capable of delivering the large 6,645-bp MYO7A cDNA....

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 37915173
DOI: 10.1016/j.ymthe.2023.10.018
PMCID: PMC10727968

Genes

MYO7A

Provenance

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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