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CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression

Huntington's disease (HD) is a fatal, neurodegenerative disorder in which patients suffer from mobility, psychological and cognitive impairments. Existing therapeutics are only symptomatic and do not significantly alter the disease progression or increase life expectancy. HD is caused by expans...

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Detalles Bibliográficos
Autores principales: Zaghloul, Eman M., Gissberg, Olof, Moreno, Pedro M.D., Siggens, Lee, Hällbrink, Mattias, Jørgensen, Anna S., Ekwall, Karl, Zain, Rula, Wengel, Jesper, Lundin, Karin E., Smith, C.I. Edvard
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5435994/
https://www.ncbi.nlm.nih.gov/pubmed/28334749
http://dx.doi.org/10.1093/nar/gkx111