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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Detalles Bibliográficos
Autores principales: Kilikevicius, Audrius, Wang, Jun, Shen, Xiulong, Rigo, Frank, Prakash, Thahza P., Napierala, Marek, Corey, David R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8928816/
https://www.ncbi.nlm.nih.gov/pubmed/35289725
http://dx.doi.org/10.1080/15476286.2022.2043650

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8928816/
https://www.ncbi.nlm.nih.gov/pubmed/35289725
http://dx.doi.org/10.1080/15476286.2022.2043650

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