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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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
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