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Article

CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression.

Nucleic acids research - 19 May 2017

Zaghloul Eman M, Gissberg Olof, Moreno Pedro M D, Siggens Lee, Hällbrink Mattias, Jørgensen Anna S, Ekwall Karl, Zain Rula, Wengel Jesper, Lundin Karin E, Smith C I Edvard

Abstract excerpt

Huntington's disease (HD) is a fatal, neurodegenerative disorder in which patients suffer from mobility, psychological and cognitive impairments. Existing therapeutics are only symptomatic and do not significantly alter the disease progression or increase life expectancy. HD is caused by expansion of the CAG trinucleotide repeat region in exon 1 of the Huntingtin gene (HTT), leading to the formation of mutant HTT...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 28334749
DOI: 10.1093/nar/gkx111
PMCID: PMC5435994

Genes

HTT

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