Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Huntington's disease (HD) is a fatal neurodegenerative disease caused by a genetic expansion of the CAG repeat region in the huntingtin (HTT) gene. Studies in HD mouse models have shown that artificial miRNAs can reduce mutant HTT, but evidence for their effectiveness and safety in larger anima...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Pfister, Edith L., DiNardo, Natalie, Mondo, Erica, Borel, Florie, Conroy, Faith, Fraser, Cara, Gernoux, Gwladys, Han, Xin, Hu, Danjing, Johnson, Emily, Kennington, Lori, Liu, PengPeng, Reid, Suzanne J., Sapp, Ellen, Vodicka, Petr, Kuchel, Tim, Morton, A. Jennifer, Howland, David, Moser, Richard, Sena-Esteves, Miguel, Gao, Guangping, Mueller, Christian, DiFiglia, Marian, Aronin, Neil
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6909722/
https://ncbi.nlm.nih.gov/pubmed/29207890
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.199
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