CRISPR/Cas9-based removal of a repeat expansion in C9ORF72 counteracts disease mechanisms
Dec. 15, 2022
The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).
