Abstract
Gene therapy for the muscular dystrophies has evolved as a promising treatment for
this progressive group of disorders. Although corticosteroids and/or supportive treatments
remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory
failure, and compromised cardiac function is the inevitable outcome. Recent developments
in genetically mediated therapies have allowed for personalized treatments that strategically
target individual muscular dystrophy subtypes based on disease pathomechanism and
phenotype. In this review, we highlight the therapeutic progress with emphasis on
evolving preclinical data and our own experience in completed clinical trials and
others currently underway. We also discuss the lessons we have learned along the way
and the strategies developed to overcome limitations and obstacles in this field.
Keywords
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Article info
Publication history
Published online: August 06, 2014
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© 2014 Elsevier Inc. Published by Elsevier Inc. All rights reserved.
