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Pre-clinical validation of a novel AAV-mediated gene therapy for <i>KCNV2</i> retinopathy improves visual function in a mouse model and expression in patient organoids

2025-12-11

Abstract excerpt

Voltage-gated (Kv) potassium channels are critical for neuronal physiology, and their dysfunction can lead to serious consequences. For example, mutations in the silent modulatory Kv8.2 subunit are known to cause irreversible inherited blindness ( KCNV2 retinopathy). This is a currently incurable condition that causes lifelong visual loss, reduced visual acuity, photoaversion, night blindness and abnormal colour...

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Literature Corpus work
79668cbb-df59-572e-bb05-80c9d18e3a70
DOI
10.64898/2025.12.09.692426
Open publication

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Pre-clinical validation of a novel AAV-mediated gene therapy for <i>KCNV2</i> retinopathy improves visual function in a mouse model and expression in patient organoidsDOI 10.64898/2025.12.09.692426
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