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 s...
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| Main Authors: | , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2015-01-01T00:00:00Z.
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