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Article

Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

20 Jun 2017

Abstract excerpt

Despite mutations in the rod phosphodiesterase 6-alpha (PDE6A) gene being well recognized as a cause of human retinitis pigmentosa, no definitive treatments have been developed to treat this blinding disease. We performed a trial of retinal gene augmentation in the Pde6a mutant dog using Pde6a delivery by capsid-mutant adeno-associated virus serotype 8, previously shown to have a rapid onset of transgene...

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PMID: 28676737
DOI: 10.3389/fnins.2017.00342

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