In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping-Reference-Cited by-同舟云学术

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

Published:2009-03 Issue:3 Volume:11 Page:257-266
ISSN:1099-498X
Container-title:The Journal of Gene Medicine
language:en
Short-container-title:J. Gene Med.

Author:

Heemskerk Hans A.,de Winter Christa L.,de Kimpe Sjef J.,van Kuik-Romeijn Petra,Heuvelmans Niki,Platenburg Gerard J.,van Ommen Gert-Jan B.,van Deutekom Judith C. T.,Aartsma-Rus Annemieke

Publisher

Wiley

Subject

Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

Reference33 articles.

1. Gene therapy: the ‘pro-sense’ approach to Duchenne muscular dystrophy;van Deutekom;Eur J Hum Genet,2005

2. Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: where are we now?;Wilton;Neuromuscul Disord,2005

3. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

4. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus;Monaco;Genomics,1988

5. Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne’s or Becker's muscular dystrophy;Hoffman;N Engl J Med,1988

Cited by 151 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. The Usefulness of Determining Plasma and Tissue Concentrations of Phosphorodiamidate Morpholino Oligonucleotides to Estimate Their Efficacy in Duchenne Muscular Dystrophy Patients;Drug Metabolism and Disposition;2024-07-11

2. Development of 2′-O-Methyl and LNA Antisense Oligonucleotides for SMN2 Splicing Correction in SMA Cells;Biomedicines;2023-11-16

3. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies;Frontiers in Physiology;2023-06-26

4. The Future of Exon Skipping for Duchenne Muscular Dystrophy;Human Gene Therapy;2023-05-01

5. A perspective on oligonucleotide therapy: Approaches to patient customization;Frontiers in Pharmacology;2022-10-19