Document Detail


Myotonic dystrophy mouse models: towards rational therapy development.
MedLine Citation:
PMID:  21724467     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
DNA repeat expansions can result in the production of toxic RNA. RNA toxicity has been best characterised in the context of myotonic dystrophy. Nearly 20 mouse models have contributed significant and complementary insights into specific aspects of this novel disease mechanism. These models provide a unique resource to test pharmacological, anti-sense, and gene-therapy therapeutic strategies that target specific events of the pathobiological cascade. Further proof-of-principle concept studies and preclinical experiments require critical and thorough analysis of the multiple myotonic dystrophy transgenic lines available. This review provides in-depth assessment of the molecular and phenotypic features of these models and their contribution towards the dissection of disease mechanisms, and compares them with the human condition. More importantly, it provides critical assessment of their suitability and limitations for preclinical testing of emerging therapeutic strategies.
Authors:
Mário Gomes-Pereira; Thomas A Cooper; Geneviève Gourdon
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Publication Detail:
Type:  Journal Article; Review     Date:  2011-07-02
Journal Detail:
Title:  Trends in molecular medicine     Volume:  17     ISSN:  1471-499X     ISO Abbreviation:  Trends Mol Med     Publication Date:  2011 Sep 
Date Detail:
Created Date:  2011-08-29     Completed Date:  2011-12-20     Revised Date:  2014-01-10    
Medline Journal Info:
Nlm Unique ID:  100966035     Medline TA:  Trends Mol Med     Country:  England    
Other Details:
Languages:  eng     Pagination:  506-17     Citation Subset:  IM    
Copyright Information:
Copyright © 2011 Elsevier Ltd. All rights reserved.
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MeSH Terms
Descriptor/Qualifier:
Alternative Splicing / genetics
Animals
CCAAT-Enhancer-Binding Protein-delta / genetics,  metabolism
DNA Repeat Expansion / genetics*
DNA-Binding Proteins / genetics,  metabolism
Disease Models, Animal*
Exons / genetics
Genetic Therapy / methods
Humans
Mice
Mice, Transgenic
Myotonic Dystrophy / genetics,  pathology*,  therapy*
RNA / metabolism
RNA-Binding Proteins / genetics,  metabolism
Grant Support
ID/Acronym/Agency:
R01 AR045653/AR/NIAMS NIH HHS; R01 AR060733/AR/NIAMS NIH HHS; R01 HL045565/HL/NHLBI NIH HHS
Chemical
Reg. No./Substance:
0/Cebpd protein, mouse; 0/DNA-Binding Proteins; 0/Mbnl1 protein, mouse; 0/RNA-Binding Proteins; 142662-43-9/CCAAT-Enhancer-Binding Protein-delta; 63231-63-0/RNA
Comments/Corrections

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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