Article
Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs.
Nucleic acid therapeutics - 1 Apr 2017
Hu Jiaxin, Rigo Frank, Prakash Thazha P, Corey David R
Abstract excerpt
Mutations within the chromosome 9 open reading frame 72 (c9orf72) gene are associated with both familial amyotrophic lateral sclerosis and frontotemporal dementia. The mutation leads to an expanded GGGGCC hexanucleotide repeat within the first intron of c9orf72 and an expanded CCCCGG repeat within a corresponding antisense transcript. Both the mutant intronic and antisense RNAs have been implicated in disease. We...
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