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Article

Non-viral gene therapy for Leber’s congenital amaurosis: progress and possibilities

20 Dec 2024

Abstract excerpt

Leber's congenital amaurosis (LCA) represents a set of rare and pervasive hereditary conditions of the retina that cause severe vision loss starting in early childhood. Targeted treatment intervention has become possible thanks to recent advances in understanding LCA genetic basis. While viral vectors have shown efficacy in gene delivery, they present challenges related to safety, low cargo capacity, and the...

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PMID: 39707712
DOI: 10.1080/17435889.2024.2443387

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