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

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...

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Enregistré dans:
Détails bibliographiques
Publié dans:Nucleic Acid Ther
Auteurs principaux: Li, Liande, Shen, Xiulong, Liu, Zhongtian, Norrbom, Michaela, Prakash, Thazha P., O'Reilly, Daniel, Sharma, Vivek K., Damha, Masad J., Watts, Jonathan K., Rigo, Frank, Corey, David R.
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc. 2018
Sujets:
Original Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5790436/
https://ncbi.nlm.nih.gov/pubmed/29341839
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2017.0703
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