Ontology highlight
ABSTRACT:
SUBMITTER: Miniarikova J
PROVIDER: S-EPMC6079476 | biostudies-literature | 2018 Apr
REPOSITORIES: biostudies-literature
Miniarikova Jana J Evers Melvin M MM Konstantinova Pavlina P
Molecular therapy : the journal of the American Society of Gene Therapy 20180208 4
The single mutation underlying the fatal neuropathology of Huntington's disease (HD) is a CAG triplet expansion in exon 1 of the huntingtin (HTT) gene, which gives rise to a toxic mutant HTT protein. There have been a number of not yet successful therapeutic advances in the treatment of HD. The current excitement in the HD field is due to the recent development of therapies targeting the culprit of HD either at the DNA or RNA level to reduce the overall mutant HTT protein. In this review, we bri ...[more]