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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Autors principals: Malerba, Alberto, Kang, Jagjeet K, McClorey, Graham, Saleh, Amer F, Popplewell, Linda, Gait, Michael J, Wood, Matthew JA, Dickson, George
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2012
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3528303/
https://ncbi.nlm.nih.gov/pubmed/23250360
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.54
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