Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency


To test whether fast-acting, self-complimentary (sc), adeno-associated virus-mediated RPE65 expression prevents cone degeneration and/or restores cone function, we studied two mouse lines: the Rpe65-deficient rd12 mouse and the Rpe65-deficient, rhodopsin null (‘that is, cone function-only’) Rpe65−/−::Rho−/− mouse. scAAV5 expressing RPE65 was injected… (More)
DOI: 10.1038/gt.2010.29

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