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Article

Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of <i>MERTK</i> Associated Retinitis Pigmentosa

1 Mar 2013

Abstract excerpt

Proof of concept for MERTK gene replacement therapy has been demonstrated using different viral vectors in the Royal College of Surgeon (RCS) rat, a well characterized model of recessive retinitis pigmentosa that contains a mutation in the Mertk gene. MERTK plays a key role in renewal of photoreceptor outer segments (OS) by phagocytosis of shed OS tips. Mutations in MERTK cause impaired phagocytic activity and...

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PMID: 23692380
DOI: 10.1089/humc.2013.037

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