Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide-Reference-Cited by-同舟云学术

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Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide

Published:2023-12 Issue: Volume:34 Page:102034
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Watanabe Naoki,Tone Yuichiro,Nagata Tetsuya,Masuda Satoru,Saito Takashi,Motohashi Norio,Takagaki Kazuchika,Aoki Yoshitsugu,Takeda Shin’ichi

Funder

Nippon Shinyaku

National Center of Neurology and Psychiatry

Japan Agency for Medical Research and Development

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference33 articles.

1. Population frequencies of inherited neuromuscular diseases--a world survey;Emery;Neuromuscul. Disord.,1991

2. The Molecular Basis for Duchenne versus Becker Muscular Dystrophy: Correlation of Severity with Type of Deletion;Koenig;Am. J. Hum. Genet.,1989

3. An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases;Rodrigues;Methods Mol. Biol.,2018

4. Golodirsen: First Approval;Heo;Drugs,2020

5. Viltolarsen: First Approval;Dhillon;Drugs,2020

Cited by 1 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. Phase I/II Trial of Brogidirsen: Dual-Targeting Antisense Oligonucleotides for Exon 44 Skipping in Duchenne Muscular Dystrophy;2024-08-30

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