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Expanded CAG/CTG repeats resist gene silencing mediated by targeted epigenome editing
Expanded CAG/CTG repeat disorders affect over 1 in 2500 individuals worldwide. Potential therapeutic avenues include gene silencing and modulation of repeat instability. However, there are major mechanistic gaps in our understanding of these processes, which prevent the rational design of an efficie...
Autores principales: | Yang, Bin, Borgeaud, Alicia C, Buřičová, Marcela, Aeschbach, Lorène, Rodríguez-Lima, Oscar, Ruiz Buendía, Gustavo A, Cinesi, Cinzia, Taylor, Alysha S, Baubec, Tuncay, Dion, Vincent |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8825355/ https://www.ncbi.nlm.nih.gov/pubmed/34494094 http://dx.doi.org/10.1093/hmg/ddab255 |
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