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Article

EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat.

PloS one - 1 Jan 2014

Zallocchi Marisa, Binley Katie, Lad Yatish, Ellis Scott, Widdowson Peter, Iqball Sharifah, Scripps Vicky, Kelleher Michelle, Loader Julie, Miskin James, Peng You-Wei, Wang Wei-Min, Cheung Linda, Delimont Duane, Mitrophanous Kyriacos A, Cosgrove Dominic

Abstract excerpt

Usher syndrome type 1B is a combined deaf-blindness condition caused by mutations in the MYO7A gene. Loss of functional myosin VIIa in the retinal pigment epithelia (RPE) and/or photoreceptors leads to blindness. We evaluated the impact of subretinally delivered UshStat, a recombinant EIAV-based lentiviral vector expressing human MYO7A, on photoreceptor function in the shaker1 mouse model for Usher type 1B that...

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Identifiers

PMID: 24705452
DOI: 10.1371/journal.pone.0094272
PMCID: PMC3976400

Genes

RPE

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