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Article

AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs

9 Feb 2016

Abstract excerpt

We previously reported that subretinal injection of AAV2/5 RK.cpde6β allowed long-term preservation of photoreceptor function and vision in the rod-cone dysplasia type 1 (rcd1) dog, a large animal model of naturally occurring PDE6β deficiency. The present study builds on these earlier findings to provide a detailed assessment of the long-term effects of gene therapy on the spatiotemporal pattern of retinal...

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PMID: 26857842
DOI: 10.1038/mt.2016.37

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