Ontology highlight
ABSTRACT:
SUBMITTER: Urbanek MO
PROVIDER: S-EPMC5368221 | biostudies-literature | 2017
REPOSITORIES: biostudies-literature
Urbanek Martyna O MO Fiszer Agnieszka A Krzyzosiak Wlodzimierz J WJ
Frontiers in cellular neuroscience 20170328
In several human polyglutamine diseases caused by expansions of CAG repeats in the coding sequence of single genes, mutant transcripts are detained in nuclear RNA foci. In polyglutamine disorders, unlike other repeat-associated diseases, both RNA and proteins exert pathogenic effects; therefore, decreases of both RNA and protein toxicity need to be addressed in proposed treatments. A variety of oligonucleotide-based therapeutic approaches have been developed for polyglutamine diseases, but conco ...[more]