Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of leber congenital amaurosis

Matthew L. Batten, Yoshikazu Imanishi, Daniel C. Tu, Thuy Doan, Li Zhu, Jijing Pang, Lyudmila Glushakova, Alexander R. Moise, Wolfgang Baehr, Russell N. Van Gelder, William W. Hauswirth, Fred Rieke, Krzysztof Palczewski

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Medicine & Life Sciences