Article
Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification.
Nucleic acid therapeutics - 1 Feb 2014
Evers Melvin M, Tran Hoang-Dai, Zalachoras Ioannis, Meijer Onno C, den Dunnen Johan T, van Ommen Gert-Jan B, Aartsma-Rus Annemieke, van Roon-Mom Willeke M C
Abstract excerpt
Huntington's disease (HD) is a progressive autosomal dominant disorder, caused by a CAG repeat expansion in the HTT gene, which results in expansion of a polyglutamine stretch at the N-terminal end of the huntingtin protein. Several studies have implicated the importance of proteolytic cleavage of mutant huntingtin in HD pathogenesis and it is generally accepted that N-terminal huntingtin fragments are more toxic...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.