AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis

<h4>Objective</h4> To evaluate efficacy of a novel adeno-associated virus (AAV) vector, AAV2/4-RS1, for retinal rescue in the retinoschisin knockout (Rs1-KO) mouse model of X-linked retinoschisis (XLRS). Brinzolamide (Azopt®), a carbonic anhydrase inhibitor, was tested for its ability to...

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Bibliographic Details
Main Authors: Brittni A. Scruggs, Sajag Bhattarai, Megan Helms, Ioana Cherascu, Adisa Salesevic, Elliot Stalter, Joseph Laird, Sheila A. Baker, Arlene V. Drack
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2022-01-01
Series:PLoS ONE
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9728878/?tool=EBI