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

OBJECTIVE: 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 potentiate the ef...

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Detalles Bibliográficos
Autores principales: Scruggs, Brittni A., Bhattarai, Sajag, Helms, Megan, Cherascu, Ioana, Salesevic, Adisa, Stalter, Elliot, Laird, Joseph, Baker, Sheila A., Drack, Arlene V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9728878/
https://www.ncbi.nlm.nih.gov/pubmed/36477475
http://dx.doi.org/10.1371/journal.pone.0276298

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