Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO-Reference-Cited by-同舟云学术

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Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Published:2010-10 Issue:10 Volume:18 Page:1822-1829
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Yin HaiFang,Moulton Hong M,Betts Corinne,Merritt Thomas,Seow Yiqi,Ashraf Shirin,Wang QingSong,Boutilier Jordan,Wood Matthew JA

Publisher

Elsevier BV

Subject

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

Reference34 articles.

1. RNA-targeted splice-correction therapy for neuromuscular disease;Wood;Brain,2010

2. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients;Aartsma-Rus;Hum Mol Genet,2003

3. Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense;Aartsma-Rus;Am J Hum Genet,2004

4. Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons;Aartsma-Rus;Mol Ther,2006

5. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology;Alter;Nat Med,2006

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1. Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle;Journal of Biosciences;2024-02-16

2. Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives;Journal of Biosciences;2023-10-11

3. Toxicity and Immune Response;CPP, Cell-Penetrating Peptides;2023

4. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy;Molecular Therapy - Nucleic Acids;2022-12

5. Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy;EMBO reports;2022-04-08

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