Ontology highlight
ABSTRACT:
SUBMITTER: Nakamori M
PROVIDER: S-EPMC7043212 | biostudies-literature | 2020 Feb
REPOSITORIES: biostudies-literature
Nature genetics 20200214 2
In many repeat diseases, such as Huntington's disease (HD), ongoing repeat expansions in affected tissues contribute to disease onset, progression and severity. Inducing contractions of expanded repeats by exogenous agents is not yet possible. Traditional approaches would target proteins driving repeat mutations. Here we report a compound, naphthyridine-azaquinolone (NA), that specifically binds slipped-CAG DNA intermediates of expansion mutations, a previously unsuspected target. NA efficiently ...[more]