Article

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

2025-04-07

Abstract excerpt

<title>Abstract</title> <p> Blue cone monochromacy (BCM) is an X-linked retinal disorder caused by mutations in the <italic>OPN1LW/OPN1MW</italic> gene locus, resulting in impaired cone function and structural degeneration. We conducted a comparative analysis of AAV-mediated gene therapy in <italic>Opn1lw/Opn1mw</italic> double knockout (DKO) and <italic>Opn1mw</italic> <sup> <italic>C198R</italic> </sup...

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Literature Corpus work: 145032f6-b6c4-5480-9308-4b101c7c5f64
DOI: 10.21203/rs.3.rs-6075007/v1

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