RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.

A hexanucleotide GGGGCC repeat expansion in the noncoding region of the C9ORF72 gene is the most common genetic abnormality in familial and sporadic amyotrophic lateral sclerosis and frontotemporal dementia . The function of the C9ORF72 protein is unknown, as is the mechanism by which the repeat expansion could cause disease.

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