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Current Pediatrics Reports
Published in: Current Pediatrics Reports 2/2014

01-06-2014 | Genetics (BR Pober, Section Editor)

Therapy of Genetic Disorders: Novel Therapies for Duchenne Muscular Dystrophy

Authors: Jane T. Seto, Niclas E. Bengtsson, Jeffrey S. Chamberlain

Published in: Current Pediatrics Reports | Issue 2/2014

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Abstract

Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therapy methods are being developed to replace the defective dystrophin gene or induce dystrophin production from mutant genes. Stem cell approaches are being developed to replace lost muscle cells while also bringing in new dystrophin genes. This review summarizes recent progress in the field with an emphasis on clinical applications.
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Metadata
Title
Therapy of Genetic Disorders: Novel Therapies for Duchenne Muscular Dystrophy
Authors
Jane T. Seto
Niclas E. Bengtsson
Jeffrey S. Chamberlain
Publication date
01-06-2014
Publisher
Springer US
Published in
Current Pediatrics Reports / Issue 2/2014
Electronic ISSN: 2167-4841
DOI
https://doi.org/10.1007/s40124-014-0044-x

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