CRISPR-mediated gene correction links the ATP7A M1311V mutations with amyotrophic lateral sclerosis pathogenesis in one individual

Yeomin Yun, Sung Ah Hong, Ka Kyung Kim, Daye Baek, Dongsu Lee, Ashwini M. Londhe, Minhyung Lee, Jihyeon Yu, Zachary T. McEachin, Gary J. Bassell, Robert Bowser, Chadwick M. Hales, Sung Rae Cho, Janghwan Kim, Ae Nim Pae, Eunji Cheong, Sangwoo Kim, Nicholas M. Boulis, Sangsu Bae, Yoon Ha

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Biochemistry, Genetics and Molecular Biology

Neuroscience

Agricultural and Biological Sciences