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Article

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9.

Gene therapy - 1 Feb 2012

Dalkara D, Byrne L C, Lee T, Hoffmann N V, Schaffer D V, Flannery J G

Abstract excerpt

Delivery of therapeutic genes to a large region of the retina with minimal damage from intraocular surgery is a central goal of treatment for retinal degenerations. Recent studies have shown that AAV9 can reach the central nervous system (CNS) and retina when administered systemically to neonates, which is a promising strategy for some retinal diseases. We investigated whether the retinal transduction efficiency...

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PMID: 22011645
DOI: 10.1038/gt.2011.163

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