A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases

Li Ou, Michael J. Przybilla, Alexandru Flaviu Tăbăran, Paula Overn, M. Gerard O’Sullivan, Xuntian Jiang, Rohini Sidhu, Pamela J. Kell, Daniel S. Ory, Chester B. Whitley

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37 Scopus citations

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Medicine & Life Sciences