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 Kell, Daniel S. Ory, Chester B. Whitley
Topics & Concepts
Sandhoff diseaseBiologyGenetic enhancementGenome editingGeneGeneticsTay-Sachs diseaseCRISPRVirologyMedicineDiseasePathologyCRISPR and Genetic EngineeringRNA regulation and diseaseVirus-based gene therapy research