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

Brittni A ScruggsSajag BhattaraiMegan HelmsIoana CherascuAdisa SalesevicElliot StalterJoseph LairdSheila A BakerArlene V Drack

Published in: PloS one (2022)

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.

Keyphrases

gene therapy
mouse model
end stage renal disease
newly diagnosed
diabetic retinopathy
chronic kidney disease
ejection fraction
optical coherence tomography
vascular endothelial growth factor
machine learning
risk assessment
patient reported
endothelial cells
climate change
wild type