Ontology highlight
ABSTRACT:
SUBMITTER: Shen X
PROVIDER: S-EPMC6129981 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Shen Xiulong X Kilikevicius Audrius A O'Reilly Daniel D Prakash Thazha P TP Damha Masad J MJ Rigo Frank F Corey David R DR
Bioorganic & medicinal chemistry letters 20180721 17
Friedreich's ataxia (FRDA) is an incurable neurodegenerative disorder caused by reduced expression of the mitochondrial protein frataxin (FXN). The genetic cause of the disease is an expanded GAA repeat within the FXN gene. Agents that increase expression of FXN protein are a potential approach to therapy. We previously described anti-trinucleotide GAA duplex RNAs (dsRNAs) and antisense oligonucleotides (ASOs) that activate FXN protein expression in multiple patient derived cell lines. Here we t ...[more]