Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

Exon skipping mediated by tricyclo-DNA (tc-DNA) antisense oligonucleotides has been shown to induce significant levels of dystrophin restoration in mdx, a mouse model of Duchenne muscular dystrophy. This translates into significant improvement in key disease indicators in muscle, cardio-respiratory...

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Bibliographic Details
Main Authors: Saleh Omairi, Kwan-Leong Hau, Henry Collin-Hooper, Federica Montanaro, Aurelie Goyenvalle, Luis Garcia, Ketan Patel
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
muscle
Duchenne
mdx
antisense oilgo
revertant
tricyclo
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117302755

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http://www.sciencedirect.com/science/article/pii/S2162253117302755

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