Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
Huntington's disease (HD) is a dominantly inherited neurodegenerative disease caused by CAG repeat expansion in exon 1 of huntingtin (HTT). Studies in mouse models of HD with a regulated mutant transgene show that continuous mutant allele expression is required for behavioral and pathological signs;...
Furkejuvvon:
| Váldodahkkit: | , , , , |
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| Materiálatiipa: | Artigo |
| Giella: | Inglês |
| Almmustuhtton: |
Elsevier
2015-01-01
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| Ráidu: | Molecular Therapy: Nucleic Acids |
| Fáttát: | |
| Liŋkkat: | http://www.sciencedirect.com/science/article/pii/S2162253116300208 |
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