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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 (Author), Xiaoyong Shen (Author), Xue Dong (Author), Ning Ran (Author), Gang Han (Author), Limin Cao (Author), Ben Gu (Author), HaiFang Yin (Author)
Format:	Book
Published:	Elsevier, 2015-01-01T00:00:00Z.
Subjects:	article
Online Access:	Connect to this object online.
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