Affordable Access

Publisher Website

Animal models of polyglutamine diseases and therapeutic approaches.

Authors
  • 1
Type
Published Article
Journal
The Journal of biological chemistry
Publication Date
Volume
284
Issue
12
Pages
7431–7435
Identifiers
DOI: 10.1074/jbc.R800065200
PMID: 18957429
Source
Medline

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.

There are no comments yet on this publication. Be the first to share your thoughts.

Statistics

Seen <100 times
0 Comments