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Animal models of polyglutamine diseases and therapeutic approaches.

Authors
  • Marsh, J Lawrence1
  • Lukacsovich, Tamas
  • Thompson, Leslie Michels
  • 1 Developmental Biology Center and the Department of Developmental and Cell Biology, University of California, Irvine, California 92697, USA. [email protected]
Type
Published Article
Journal
The Journal of biological chemistry
Publication Date
Mar 20, 2009
Volume
284
Issue
12
Pages
7431–7435
Identifiers
DOI: 10.1074/jbc.R800065200
PMID: 18957429
Source
Medline
License
Unknown

Abstract

The dominant gain-of-function polyglutamine repeat diseases, in which the initiating mutation is known, allow development of models that recapitulate many aspects of human disease. To the extent that pathology is a consequence of disrupted fundamental cellular activities, one can effectively study strategies to ameliorate or protect against these cellular insults. Model organisms allow one to identify pathways that affect disease onset and progression, to test and screen for pharmacological agents that affect pathogenic processes, and to validate potential targets genetically as well as pharmacologically. Here, we describe polyglutamine repeat diseases that have been modeled in a variety of organisms, including worms, flies, mice, and non-human primates, and discuss examples of how they have broadened the therapeutic landscape.

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