Ontology highlight
ABSTRACT:
SUBMITTER: Rindt H
PROVIDER: S-EPMC5641306 | biostudies-literature | 2017
REPOSITORIES: biostudies-literature
Rindt Hansjörg H Tom Colton M CM Lorson Christian L CL Mattis Virginia B VB
Frontiers in neuroscience 20171010
Huntington's disease (HD) is a devastating neurodegenerative disorder caused by a polyglutamine (polyQ) expansion in exon 1 of the <i>Huntingtin</i> (<i>HTT</i>) gene. We have previously demonstrated that spliceosome-mediated <i>trans</i>-splicing is a viable molecular strategy to specifically reduce and repair mutant HTT (mtHTT). Here, the targeted tethering efficacy of the pre-mRNA <i>trans</i>-splicing modules (PTM) in HTT was optimized. Various PTMs that targeted the 3' end of HTT intron 1 o ...[more]