G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects

Friedreich’s ataxia is an incurable disease caused by frataxin (FXN) protein deficiency, which is mostly induced by GAA repeat expansion in intron 1 of the FXN gene. Here, we identified antisense oligonucleotides (ASOs), complementary to two regions within the first intron of FXN pre-mRNA, which cou...

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Detalles Bibliográficos
Autores principales: Wang, Feng, Calvo-Roitberg, Ezequiel, Rembetsy-Brown, Julia M, Fang, Minggang, Sousa, Jacquelyn, Kartje, Zachary J, Krishnamurthy, Pranathi Meda, Lee, Jonathan, Green, Michael R, Pai, Athma A, Watts, Jonathan K
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9825156/
https://www.ncbi.nlm.nih.gov/pubmed/36511872
http://dx.doi.org/10.1093/nar/gkac1108

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9825156/
https://www.ncbi.nlm.nih.gov/pubmed/36511872
http://dx.doi.org/10.1093/nar/gkac1108

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