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» CRISPR/Cas9-based removal of a repeat expansion in C9ORF72 counteracts disease mechanisms
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Neurology/Psychiatric
CRISPR/Cas9-based removal of a repeat expansion in C9ORF72 counteracts disease mechanisms
Dec. 15, 2022
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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).
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Neurology/psychiatric