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Article

Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.

Biochemistry - 22 Jul 2014

Hu Jiaxin, Liu Jing, Narayanannair K Jayaprakash, Lackey Jeremy G, Kuchimanchi Satya, Rajeev Kallanthottathil G, Manoharan Muthiah, Swayze Eric E, Lima Walt F, Prakash Thazha P, Xiang Qin, Martinez Carlos, Corey David R

Abstract excerpt

Dentatorubral-pallidoluysian atrophy (DRPLA) is a progressive neurodegenerative disorder that currently has no curative treatments. DRPLA is caused by an expansion of a CAG trinucleotide repeat region within the protein-encoding sequence of the atrophin-1 (ATN-1) gene. Inhibition of mutant ATN-1 protein expression is one strategy for treating DRPLA, and allele-selective gene silencing agents that block mutant...

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Identifiers

PMID: 24981774
DOI: 10.1021/bi500610r
PMCID: PMC4108179

Genes

HTT

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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