Document Detail

Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.
MedLine Citation:
PMID:  23387802     Owner:  NLM     Status:  MEDLINE    
Muscular dystrophies are genetic disorders characterized by skeletal muscle wasting and weakness. Although there is no effective therapy, a number of experimental strategies have been developed over recent years and some of them are undergoing clinical investigation. In this review, we highlight recent developments and key challenges for strategies based upon gene replacement and gene/expression repair, including exon-skipping, vector-mediated gene therapy and cell therapy. Therapeutic strategies for different forms of muscular dystrophy are discussed, with an emphasis on Duchenne muscular dystrophy, given the severity and the relatively advanced status of clinical studies for this disease.
Sara Benedetti; Hidetoshi Hoshiya; Francesco Saverio Tedesco
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't; Review     Date:  2013-03-04
Journal Detail:
Title:  The FEBS journal     Volume:  280     ISSN:  1742-4658     ISO Abbreviation:  FEBS J.     Publication Date:  2013 Sep 
Date Detail:
Created Date:  2013-08-20     Completed Date:  2013-10-24     Revised Date:  2014-02-20    
Medline Journal Info:
Nlm Unique ID:  101229646     Medline TA:  FEBS J     Country:  England    
Other Details:
Languages:  eng     Pagination:  4263-80     Citation Subset:  IM    
Copyright Information:
© 2013 The Authors Journal compilation © 2013 FEBS.
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MeSH Terms
Cell- and Tissue-Based Therapy*
Genetic Therapy*
Muscular Dystrophy, Duchenne / genetics*,  therapy*
Stem Cell Transplantation*
Stem Cells / cytology*,  metabolism
Grant Support
MR/J006785/1//Medical Research Council; //Medical Research Council

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