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Article

Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

13 Oct 2009

Abstract excerpt

The ability of AAV2/5 to target murine photoreceptors was used to optimize vector expression cassettes for rhodopsin replacement gene therapy in the retina. Defects in this pivotal protein in the visual cycle are associated with various forms of inherited eye defects including retinitis pigmentosa. A final optimized vector design led to functional correction and regeneration of rod outer segment in a mouse model...

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PMID: 19824806
DOI: 10.1089/hum.2009.119

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