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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Pubblicato in:Hum Gene Ther
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2018
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Research Articles
Accesso online: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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