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

Authors: 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 (Source: Gene Therapy)

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