Aav2/4-Rs1 Gene Therapy In The Retinoschisin Knockout Mouse Model Of X-Linked Retinoschisis

Brittni A. Scruggs,Sajag Bhattarai,Ioana Cherascu,Megan Helms,Adisa Salesevic,Joseph Laird,Sheila A. Baker,Budd Tucker,Arlene V. Drack

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE（2019）

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摘要

AAV2/4-RS1 shows promise for improving retinal phenotype in the Rs1-KO mouse model. Subretinal delivery was superior to intravitreal. Topical brinzolamide did not improve efficacy. AAV2/4-RS1 may be considered as a potential treatment for XLRS patients.

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