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Article

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

22 Jun 2011

Abstract excerpt

Gene therapy is emerging as a therapeutic modality for treating disorders of the retina. Photoreceptor cells are the primary cell type affected in many inherited diseases of retinal degeneration. Successfully treating these diseases with gene therapy requires the identification of efficient and safe targeting vectors that can transduce photoreceptor cells. One serotype of adeno-associated virus, AAV2, has been...

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PMID: 21697530
DOI: 10.1126/scitranslmed.3002103

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