Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

The knockdown of myostatin, a negative regulator of skeletal muscle mass may have important implications in disease conditions accompanied by muscle mass loss like cancer, HIV/AIDS, sarcopenia, muscle atrophy, and Duchenne muscular dystrophy (DMD). In DMD patients, where major muscle loss has occurr...

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Bibliographic Details
Main Authors: Alberto Malerba, Jagjeet K Kang, Graham McClorey, Amer F Saleh, Linda Popplewell, Michael J Gait, Matthew JA Wood, George Dickson
Format: Article
Language:English
Published: Elsevier 2012-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotides
Duchenne muscular dystrophy
dystrophin
exon skipping
myostatin
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116301202

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