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

J. Pang, S. E. Boye, B. Lei, S. L. Boye, D. Everhart, R. Ryals, Y. Umino, B. Rohrer, J. Alexander, J. Li, X. Dai, Q. Li, B. Chang, R. Barlow, W. W. Hauswirth

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Medicine & Life Sciences