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RNA therapy for polyglutamine neurodegenerative diseases.

Abstract:

Polyglutamine neurodegenerative diseases result from the expansion of a trinucleotide CAG repeat, encoding a polyglutamine tract in the disease-causing protein. The process by which each polyglutamine protein exerts its toxicity is complex, involving a variety of mechanisms including transcriptional dysregulation, proteasome impairment and mitochondrial dysfunction. Thus, the most effective and widely applicable therapies are likely to be those designed to eliminate production of the mutant p...

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Publisher copy:
10.1017/erm.2011.1

Authors


Watson, LM More by this author
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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Journal:
Expert reviews in molecular medicine
Volume:
14
Pages:
e3
Publication date:
2012
DOI:
EISSN:
1462-3994
ISSN:
1462-3994
URN:
uuid:0949482a-8361-4755-8e2d-fe93d1710866
Source identifiers:
245896
Local pid:
pubs:245896

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