Optimization of Antisense Oligonucleotide Drugs for Skipping Dystrophin Exon 51 and 53 in Duchenne Muscular Dystrophy

被引：0
|
作者：
Wu, Bo [1 ]
机构：
[1] Carolinas Med Ctr, Charlotte, NC 28203 USA
来源：
MOLECULAR THERAPY | 2018年 / 26卷 / 05期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
315
引用
收藏
页码：148 / 148
页数：1
相关论文
共 50 条
  • [41] Exon-Skipping in Duchenne Muscular Dystrophy
    Takeda, Shin ichi
    Clemens, Paula R.
    Hoffman, Eric P.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2021, 8 : S343 - S358
  • [42] The Future of Exon Skipping for Duchenne Muscular Dystrophy
    Aartsma-Rus, Annemieke
    HUMAN GENE THERAPY, 2023, 34 (9-10) : 372 - 378
  • [43] Exon skipping therapy for Duchenne muscular dystrophy
    Kole, Ryszard
    Krieg, Arthur M.
    ADVANCED DRUG DELIVERY REVIEWS, 2015, 87 : 104 - 107
  • [44] Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular Dystrophy
    Matsuo, Masafumi
    JMA JOURNAL, 2021, 4 (03): : 232 - 240
  • [45] Predictors of requiring port-a-cath placement in boys with Duchenne muscular dystrophy on antisense oligonucleotide exon skipping therapy
    Wright, S.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S174 - S174
  • [46] Exon 53 skipping of the human dystrophin transcript with different chemically modified AONs in a mouse model for Duchenne muscular dystrophy
    Engelbeen, Sarah
    O'Reilly, Daniel
    Van De Vijver, Davy
    Verhaart, Ingrid
    van Putten, Maaike
    Hariharan, Vignesh
    Khvorova, Anastasia
    Aartsma-Rus, Annemieke
    Damha, Masad
    EUROPEAN JOURNAL OF HUMAN GENETICS, 2024, 32 : 515 - 516
  • [47] Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy
    Yokota, Toshifumi
    Pistilli, Emidio
    Duddy, William
    Nagaraju, Kanneboyina
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2007, 7 (06) : 831 - 842
  • [48] Towards clinical studies on antisense-mediated exon skipping in Duchenne Muscular Dystrophy
    van Ommen, Gert-Jan B.
    Heemskerk, Hans
    Aartsma-Rus, Annemieke
    de Winter, Christa
    van Kuik, Petra
    de Kimpe, Sjef
    Platenburg, Gerard
    van Deutekom, Judith C. T.
    NEUROMUSCULAR DISORDERS, 2006, 16 : S95 - S95
  • [49] Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
    Aartsma-Rus, Annemieke
    Fokkema, Ivo
    Verschuuren, Jan
    Ginjaar, Leke
    van Deutekom, Judith
    van Ommen, Gert-Jan
    den Dunnen, Johan T.
    HUMAN MUTATION, 2009, 30 (03) : 293 - 299
  • [50] ANTISENSE-BASED EXON SKIPPING AS A THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY, STATUS AND PROSPECTS
    van Ommen, G. -J.
    Aartsma-Rus, A.
    Evers, M.
    van Roon, W.
    'tHoen, P. -B.
    Kemaladewi, D.
    Verbeek, S.
    Yllmaz-Elis, S.
    Dauwerse, H.
    Lesnik-Oberstein, S.
    den Dunnen, J. T.
    Goemans, N.
    Tulinus, M.
    Verschuuren, J.
    de Kimpe, S.
    Campion, G.
    van Deutekom, J.
    EUROPEAN JOURNAL OF NEUROLOGY, 2011, 18 : 7 - 7
12345