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Article

Hsp40 gene therapy exerts therapeutic effects on polyglutamine disease mice via a non-cell autonomous mechanism.

PloS one - 1 Jan 2012

Popiel H Akiko, Takeuchi Toshihide, Fujita Hiromi, Yamamoto Kazuhiro, Ito Chiyomi, Yamane Hiroshi, Muramatsu Shin-ichi, Toda Tatsushi, Wada Keiji, Nagai Yoshitaka

Abstract excerpt

The polyglutamine (polyQ) diseases such as Huntington's disease (HD), are neurodegenerative diseases caused by proteins with an expanded polyQ stretch, which misfold and aggregate, and eventually accumulate as inclusion bodies within neurons. Molecules that inhibit polyQ protein misfolding/aggregation, such as Polyglutamine Binding Peptide 1 (QBP1) and molecular chaperones, have been shown to exert therapeutic...

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Identifiers

PMID: 23226463
DOI: 10.1371/journal.pone.0051069
PMCID: PMC3511362

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