AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype-Reference-Cited by-同舟云学术

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AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype

Published:2004-11 Issue:5 Volume:10 Page:821-828
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Yoshimura Madoka,Sakamoto Miki,Ikemoto Madoka,Mochizuki Yasushi,Yuasa Katsutoshi,Miyagoe-Suzuki Yuko,Takeda Shin'ichi

Publisher

Elsevier BV

Subject

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

Reference28 articles.

1. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs;Acsadi;Nature,1991

2. Dystrophin expression in muscle following gene transfer with a fully deleted (“gutted”)adenovirus is markedly improved by trans-acting adenoviral gene products;Gilbert;Hum. Gene Ther.,2001

3. Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin;Dello Russo;Proc. Natl. Acad. Sci. USA,2002

4. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse;Lu;Nat. Med.,2003

5. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide;Bartlett;Nat. Biotechnol.,2000

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1. Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials;Human Gene Therapy;2023-05-01

2. Use of Mesenchymal Stem Cells to Enhance the Efficacy of Gene Therapy;Methods in Molecular Biology;2022-11-19

3. Development of Therapeutic RNA Manipulation for Muscular Dystrophy;Frontiers in Genome Editing;2022-05-10

4. Gene Transfer of Skeletal Muscle-Type Myosin Light Chain Kinase via Adeno-Associated Virus 6 Improves Muscle Functions in an Amyotrophic Lateral Sclerosis Mouse Model;International Journal of Molecular Sciences;2022-02-03

5. Therapeutic Application of Extracellular Vesicles-Capsulated Adeno-Associated Virus Vector via nSMase2/Smpd3, Satellite, and Immune Cells in Duchenne Muscular Dystrophy;International Journal of Molecular Sciences;2022-01-28

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