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Article

[Gene silencing approaches for the treatment of Huntington's disease].

Medecine sciences : M/S - 1 Feb 2015

Merienne Nicolas, Déglon Nicole

Abstract excerpt

Huntington's disease is a rare neurodegenerative disease caused by a pathologic CAG expansion in the exon 1 of the huntingtin (HTT) gene. Aggregation and abnormal function of the mutant HTT (mHTT) cause motor, cognitive and psychiatric symptoms in patients, which lead to death in 15-20 years. Currently, there is no treatment for HD. Experimental approaches based on drug, cell or gene therapy are developed and...

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Identifiers

PMID: 25744262
DOI: 10.1051/medsci/20153102012

Genes

HTT

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