Article

Molecular Mechanisms Limiting the Therapeutic Window of AAV Gene Therapy in Mouse Models of Blue Cone Monochromacy

2025-02-16

Abstract excerpt

<h4>ABSTRACT</h4> Blue cone monochromacy (BCM) is an X-linked retinal disorder caused by mutations in the OPN1LW/OPN1MW gene locus, resulting in impaired cone function and structural degeneration. We conducted a comparative analysis of AAV-mediated gene therapy in Opn1lw/Opn1mw double knockout (DKO) and Opn1mw C198R /Opn1sw -/- (C198R) BCM mouse models and evaluated the therapeutic window, efficacy, and long...

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Literature Corpus work: 249c718f-615c-5c95-a535-51d50ba9e3f5
DOI: 10.1101/2025.02.14.638316

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