Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

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Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure with drisapersen, an AO candidate drug in phase 3 trial, highlights the importance of exploring other effective AO chemistries for DMD. Previ...

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Bibliographic Details
Main Authors: Xianjun Gao, Xiaoyong Shen, Xue Dong, Ning Ran, Gang Han, Limin Cao, Ben Gu, HaiFang Yin
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
Duchenne muscular dystrophy
dystrophin
exon skipping
peptide nucleic acid
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116300403

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http://www.sciencedirect.com/science/article/pii/S2162253116300403

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