Article
Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.
Nucleic acid therapeutics - 1 Apr 2013
Leger Andrew J, Mosquea Leocadia M, Clayton Nicholas P, Wu I-Huan, Weeden Timothy, Nelson Carol A, Phillips Lucy, Roberts Errin, Piepenhagen Peter A, Cheng Seng H, Wentworth Bruce M
Abstract excerpt
Expansions of CUG trinucleotide sequences in RNA transcripts provide the basis for toxic RNA gain-of-function that leads to detrimental changes in RNA metabolism. A CTG repeat element normally resides in the 3' untranslated region of the dystrophia myotonica-protein kinase (DMPK) gene, but when expanded it is the genetic lesion of myotonic dystrophy type 1 (DM1), a hereditary neuromuscular disease. The pathogenic...
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