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Article

Tripartite AAV Systems for <i>EYS</i> Retinal Gene Therapy

2025-12-05

Abstract excerpt

<h4>ABSTRACT</h4> Mutations in the Eyes Shut Homolog ( EYS ) gene are a leading cause of autosomal recessive retinitis pigmentosa, a progressive retinal degenerative disease for which no effective treatment currently exists. However, the large size of the EYS coding sequence (∼9.4 kb) exceeds the packaging limit of adeno-associated virus (AAV) vectors, posing a major barrier to gene replacement therapy. To add...

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Identifiers and source

Literature Corpus work
8701a459-c205-5251-b440-e5082c2bd274
DOI
10.64898/2025.12.03.692187
Open publication

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Tripartite AAV Systems for <i>EYS</i> Retinal Gene TherapyDOI 10.64898/2025.12.03.692187
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