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

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 len...

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Главные авторы:	Marisa Zallocchi, Katie Binley, Yatish Lad, Scott Ellis, Peter Widdowson, Sharifah Iqball, Vicky Scripps, Michelle Kelleher, Julie Loader, James Miskin, You-Wei Peng, Wei-Min Wang, Linda Cheung, Duane Delimont, Kyriacos A Mitrophanous, Dominic Cosgrove
Формат:	Artigo
Язык:	Inglês
Опубликовано:	Public Library of Science (PLoS) 2014-01-01
Серии:	PLoS ONE
Online-ссылка:	https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0094272&type=printable
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