The significance of exons skipping therapy in the treatment of Duchenne muscular dystrophy

被引:0
|
作者
Takeda, Shinichi [1 ,2 ]
机构
[1] Natl Ctr Neurol & Psychiat, Translat Med Ctr, Kodaira, Tokyo 1878502, Japan
[2] Natl Ctr Neurol & Psychiat, Natl Inst Neurosci, Dept Mol Therapy, Kodaira, Tokyo 1878502, Japan
关键词
D O I
10.1016/j.neures.2010.07.008
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
引用
收藏
页码:E53 / E53
页数:1
相关论文
共 50 条
  • [21] The Future of Exon Skipping for Duchenne Muscular Dystrophy
    Aartsma-Rus, Annemieke
    HUMAN GENE THERAPY, 2023, 34 (9-10) : 372 - 378
  • [22] RNA Targeting Gene Therapy Exon-Skipping Approach for the Treatment of Duchenne Muscular Dystrophy
    Lardelli, Rea
    Roth, Daniela Martino
    Geddes, Claire
    Nachtrab, Greg
    Wilson, Alistair
    Lee, Shawn
    Ross, Olivia
    Negraes, Priscilla Davidson
    Gibbs, Dan
    Zisoulis, Dimitrios
    Batra, Ranjan
    MOLECULAR THERAPY, 2021, 29 (04) : 201 - 201
  • [23] Eteplirsen treatment for Duchenne muscular dystrophy Exon skipping and dystrophin production
    Charleston, Jay S.
    Schnell, Frederick J.
    Dworzak, Johannes
    Donoghue, Cas
    Lewis, Sarah
    Chen, Lei
    Young, G. David
    Milici, Anthony J.
    Voss, Jon
    DeAlwis, Uditha
    Wentworth, Bruce
    Rodino-Klapac, Louise R.
    Sahenk, Zarife
    Frank, Diane
    Mendell, Jerry R.
    NEUROLOGY, 2018, 90 (24) : E2146 - E2154
  • [24] Questions about efficacy of exon-skipping therapy for duchenne muscular dystrophy
    Poduri, Annapurna
    Salih, Mustafa
    Walsh, Christopher A.
    ANNALS OF NEUROLOGY, 2014, 75 (02) : 326 - 326
  • [25] Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy
    Nakamura, Akinori
    JOURNAL OF HUMAN GENETICS, 2017, 62 (10) : 871 - 876
  • [26] Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy
    Akinori Nakamura
    Journal of Human Genetics, 2017, 62 : 871 - 876
  • [27] Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy
    Aartsma-Rus, A
    Bremmer-Bout, M
    Janson, AAM
    den Dunnen, JT
    van Ommen, GJB
    van Deutekom, JCT
    NEUROMUSCULAR DISORDERS, 2002, 12 : S71 - S77
  • [28] Pip peptide conjugates of exon skipping PMO for therapy of Duchenne muscular dystrophy
    Arzumanov, Liz O'Donovan Andrey A.
    Gait, Michael J.
    HUMAN GENE THERAPY, 2014, 25 (11) : A13 - A14
  • [29] Dual exon skipping in myostatin and dystrophin as a potential therapy for Duchenne muscular dystrophy
    Kemaladewi, D. U.
    Hoogaars, W. M. H.
    van Heiningen, S. H.
    van Deutekom, J. C. T.
    den Dunnen, J. T.
    van Ommen, G. J. B.
    Aartsma-Rus, A. R.
    ten Dijke, P.
    't Hoen, P. A. C.
    NEUROMUSCULAR DISORDERS, 2009, 19 (8-9) : 577 - 577
  • [30] Exon-skipping advances for Duchenne muscular dystrophy
    Echevarria, Lucia
    Aupy, Philippine
    Goyenvalle, Aurelie
    HUMAN MOLECULAR GENETICS, 2018, 27 (R2) : R163 - R172