Document Detail


Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy.
MedLine Citation:
PMID:  19416897     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by mutations in the gene encoding dystrophin. Loss of dystrophin results in reduced sarcolemmal integrity and increased susceptibility to muscle damage. The alpha(7)beta(1)-integrin is a laminin-binding protein up-regulated in the skeletal muscle of DMD patients and in the mdx mouse model. Transgenic overexpression of the alpha(7)-integrin alleviates muscle disease in dystrophic mice, making this gene a target for pharmacological intervention. Studies suggest laminin may regulate alpha(7)-integrin expression. To test this hypothesis, mouse and human myoblasts were treated with laminin and assayed for alpha(7)-integrin expression. We show that laminin-111 (alpha(1), beta(1), gamma(1)), which is expressed during embryonic development but absent in normal or dystrophic skeletal muscle, increased alpha(7)-integrin expression in mouse and DMD patient myoblasts. Injection of laminin-111 protein into the mdx mouse model of DMD increased expression of alpha(7)-integrin, stabilized the sarcolemma, restored serum creatine kinase to wild-type levels, and protected muscle from exercised-induced damage. These findings demonstrate that laminin-111 is a highly potent therapeutic agent for the mdx mouse model of DMD and represents a paradigm for the systemic delivery of extracellular matrix proteins as therapies for genetic diseases.
Authors:
Jachinta E Rooney; Praveen B Gurpur; Dean J Burkin
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural     Date:  2009-04-28
Journal Detail:
Title:  Proceedings of the National Academy of Sciences of the United States of America     Volume:  106     ISSN:  1091-6490     ISO Abbreviation:  Proc. Natl. Acad. Sci. U.S.A.     Publication Date:  2009 May 
Date Detail:
Created Date:  2009-05-13     Completed Date:  2009-06-22     Revised Date:  2010-09-27    
Medline Journal Info:
Nlm Unique ID:  7505876     Medline TA:  Proc Natl Acad Sci U S A     Country:  United States    
Other Details:
Languages:  eng     Pagination:  7991-6     Citation Subset:  IM    
Affiliation:
Department of Pharmacology, University of Nevada School of Medicine, Reno, NV 89557, USA.
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MeSH Terms
Descriptor/Qualifier:
Animals
Antigens, CD / genetics*,  metabolism
Cell Separation
Creatine Kinase / blood
Extracellular Matrix / metabolism
Humans
Integrin alpha Chains / genetics*,  metabolism
Laminin / genetics*,  metabolism,  physiology*
Mice
Mice, Inbred C57BL
Mice, Inbred mdx
Mice, Transgenic
Models, Biological
Muscles / metabolism
Muscular Dystrophy, Duchenne / genetics*,  metabolism
Promoter Regions, Genetic
Grant Support
ID/Acronym/Agency:
R01AR053697/AR/NIAMS NIH HHS; R21NS058429/NS/NINDS NIH HHS
Chemical
Reg. No./Substance:
0/Antigens, CD; 0/Integrin alpha Chains; 0/Laminin; 0/integrin alpha7; EC 2.7.3.2/Creatine Kinase
Comments/Corrections
Erratum In:
Proc Natl Acad Sci U S A. 2009 Sep 8;106(36):15514

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


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