Difficulties translating antisense-mediated activation of Frataxin expression from cell culture to mice

Difficulties translating antisense-mediated activation of Frataxin expression from cell culture to mice

Friedreich’s ataxia (FA) is an inherited neurodegenerative disorder caused by decreased expression of frataxin (FXN) protein. Previous studies have shown that antisense oligonucleotides (ASOs) and single-stranded silencing RNAs can be used to increase expression of frataxin in cultured patient-deriv...

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Bibliographic Details
Main Authors: Audrius Kilikevicius, Jun Wang, Xiulong Shen, Frank Rigo, Thahza P. Prakash, Marek Napierala, David R. Corey
Format: Article
Language:English
Published: Taylor & Francis Group 2022-12-01
Series:RNA Biology
Subjects:
friedrich’s ataxia; frataxin; antisense oligonucleotide; trinucleotide repeat
Online Access:http://dx.doi.org/10.1080/15476286.2022.2043650

Internet

http://dx.doi.org/10.1080/15476286.2022.2043650

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