Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice-Reference-Cited by-同舟云学术

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Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice

Published:2014-07 Issue:7 Volume:22 Page:1333-1341
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Gao Xianjun,Zhao Jingwen,Han Gang,Zhang Yajie,Dong Xue,Cao Limin,Wang Qingsong,Moulton Hong M,Yin HaiFang

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference29 articles.

1. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study;Cirak;Lancet,2011

2. Systemic administration of PRO051 in Duchenne's muscular dystrophy;Goemans;N Engl J Med,2011

3. Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice;Yin;Mol Ther,2008

4. Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse;Yin;Mol Ther,2010

5. Locked nucleic acids: a promising molecular family for gene-function analysis and antisense drug development;Orum;Curr Opin Mol Ther,2001

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