Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat.

@article{Li2018ActivationOF,
  title={Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat.},
  author={Liande Li and Xiulong Shen and Zhongtian Liu and Michaela Norrbom and Thazha P. Prakash and Daniel O'Reilly and Vivek Kumar Sharma and Masad J Damha and Jonathan K Watts and Frank W Rigo and David R. Corey},
  journal={Nucleic acid therapeutics},
  year={2018},
  volume={28 1},
  pages={23-33}
}
Friedreich's Ataxia (FA) is an inherited neurologic disorder caused by an expanded GAA repeat within intron 1 of the frataxin (FXN) gene that reduces expression of FXN protein. Agents that increase expression of FXN have the potential to alleviate the disease. We previously reported that duplex RNAs (dsRNAs) and antisense oligonucleotides (ASOs) complementary to the GAA repeat could enhance expression of FXN protein. We now explore the potential of a diverse group of chemically modified dsRNAs… CONTINUE READING
Related Discussions
This paper has been referenced on Twitter 10 times. VIEW TWEETS