Ontology highlight
ABSTRACT:
SUBMITTER: Dabrowska M
PROVIDER: S-EPMC5834764 | biostudies-literature | 2018
REPOSITORIES: biostudies-literature
Dabrowska Magdalena M Juzwa Wojciech W Krzyzosiak Wlodzimierz J WJ Olejniczak Marta M
Frontiers in neuroscience 20180226
Huntington's disease (HD) is a progressive autosomal dominant neurodegenerative disorder caused by the expansion of CAG repeats in the first exon of the huntingtin gene (<i>HTT</i>). The accumulation of polyglutamine-rich huntingtin proteins affects various cellular functions and causes selective degeneration of neurons in the striatum. Therapeutic strategies used to date to silence the expression of mutant <i>HTT</i> include antisense oligonucleotides, RNA interference-based approaches and, rec ...[more]