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Slipped-CAG DNA binding small molecule induces trinucleotide repeat contractions in vivo
In many repeat diseases, like Huntington’s disease (HD), ongoing repeat expansions in affected tissues contribute to disease onset, progression and severity. Inducing contractions of expanded repeats by exogenous agents is not yet possible. Traditional approaches would target proteins driving repeat...
Autores principales: | Nakamori, Masayuki, Panigrahi, Gagan B., Lanni, Stella, Gall-Duncan, Terence, Hayakawa, Hideki, Tanaka, Hana, Luo, Jennifer, Otabe, Takahiro, Li, Jinxing, Sakata, Akihiro, Caron, Marie-Christine, Joshi, Niraj, Prasolava, Tanya, Chiang, Karen, Masson, Jean-Yves, Wold, Marc S., Wang, Xiaoxiao, Lee, Marietta Y. W. T., Huddleston, John, Munson, Katherine M., Davidson, Scott, Layeghifard, Mehdi, Edward, Lisa-Monique, Gallon, Richard, Santibanez-Koref, Mauro, Murata, Asako, Takahashi, Masanori P., Eichler, Evan E., Shlien, Adam, Nakatani, Kazuhiko, Mochizuki, Hideki, Pearson, Christopher E. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7043212/ https://www.ncbi.nlm.nih.gov/pubmed/32060489 http://dx.doi.org/10.1038/s41588-019-0575-8 |
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