Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that primarily affects the motor system, resulting in progressive muscle weakness and — ultimately — death. The disease ...

Here, the authors develop a high-fidelity RNA-targeting CRISPR system to suppress the repeat RNA expressed from the C9ORF72 gene, which improved abnormalities in a mouse model. CRISPR-CasRx ...