Article

Gene-Agnostic Gene Therapy Strategies for Inherited Retinal Diseases: Neuroprotection and Immunomodulation Beyond Genetic Correction

2026-01-09

Abstract excerpt

<h4>Background: </h4> /Objectives: Inherited retinal diseases (IRDs) represent a genetically heterogeneous group of disorders caused by mutations in over 280 genes with more than 3,100 identified variants. While gene-specific replacement therapies have achieved landmark success with voretigene neparvovec (Luxturna) for RPE65-associated retinal dystrophy, developing individual therapies for each genetic subtype rem...

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Literature Corpus work: 4325aeed-4d29-5a72-b7ae-f955a7c6dc10
DOI: 10.20944/preprints202601.0607.v1

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