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CHANGES IN RETINAL SENSITIVITY AFTER GENE THERAPY IN CHOROIDEREMIA.

08:00 EDT 9th October 2018 | BioPortfolio

Summary of "CHANGES IN RETINAL SENSITIVITY AFTER GENE THERAPY IN CHOROIDEREMIA."

Choroideremia (CHM) is a rare inherited retinal degeneration resulting from mutation of the CHM gene, which results in absence of functional Rab escort protein 1 (REP1). We evaluated retinal gene therapy with an adeno-associated virus vector that used to deliver a functional version of the CHM gene (AAV2-REP1).

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This article was published in the following journal.

Name: Retina (Philadelphia, Pa.)
ISSN: 1539-2864
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Medical and Biotech [MESH] Definitions

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