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

Gene Therapy, Published online: 02 January 2020; doi:10.1038/s41434-019-0120-5A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases

Source: Gene Therapy - January 1, 2020 Category: Genetics & Stem Cells Authors: Li Ou Michael J. Przybilla Alexandru-Flaviu T ăbăran Paula Overn M. Gerard O ’Sullivan Xuntian Jiang Rohini Sidhu Pamela J. Kell Daniel S. Ory Chester B. Whitley Source Type: research