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

作者：Li Ou, Michael J. Przybilla, Alexandru-Flaviu Tăbăran, Paula Overn, M. Gerard O’Sullivan, Xuntian Jiang, Rohini Sidhu, Pamela Kell, Daniel S. Ory, Chester B. Whitley · 发表于：Gene Therapy · 年份：2020 · DOI：10.1038/s41434-019-0120-5 · 被引用次数：54 · 研究领域：CRISPR and Genetic Engineering、RNA regulation and disease、Virus-based gene therapy research