Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa.

Mol Ther · 2017 · PMC5542800 · PMID 28549772

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Rpgr −/y mice also showed a significant rescue of cone function under light-adapted conditions, whereas the responses in C57BL/6J Rd9/Boc mice showed a trend for efficacy, which did not reach the significance level in light-adapted conditions at this time point.

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