Effect of different dosing regimens of exon skipping antisense oligonucleotides on functional outcome in the mdx mouse model of Duchenne muscular dystrophy

被引：0
|
作者：
Datson, N. [1 ]
Weij, R. [1 ]
Bijl, S. [1 ]
Janson, A. [1 ]
Vermue, R. [1 ]
Testerink, J. [1 ]
van Deutekom, J. [1 ]
机构：
[1] BioMarin, Leiden, Netherlands
来源：
NEUROMUSCULAR DISORDERS | 2015年 / 25卷
关键词：
D O I：
10.1016/j.nmd.2015.06.375
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
G.P.351
引用
收藏
页码：S291 / S291
页数：1
相关论文
共 50 条
  • [21] Evaluation of exon skipping activity of 2′-deoxy-2′-fluoro antisense oligonucleotides for Duchenne muscular dystrophy
    Jirka, S. M. G.
    van der Meulen, J. W.
    Tanganyika-de Winter, C. L.
    van Putten, M.
    Hiller, M.
    Vermue, R.
    de Visser, P. C.
    Aartsma-Rus, A.
    NEUROMUSCULAR DISORDERS, 2014, 24 (9-10) : 827 - 828
  • [22] An opening in Duchenne muscular dystrophy:: persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice
    Goyenvalle, A
    Vulin, A
    Fougerousse, F
    Leturcq, F
    Kaplan, JC
    Garcia, L
    Danos, O
    M S-MEDECINE SCIENCES, 2004, 20 (12): : 1163 - 1165
  • [23] Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy
    Echigoya, Yusuke
    Lim, Kenji Rowel Q.
    Trieu, Nhu
    Bao, Bo
    Nichols, Bailey Miskew
    Vila, Maria Candida
    Novak, James S.
    Hara, Yuko
    Lee, Joshua
    Touznik, Aleksander
    Mamchaoui, Kamel
    Aoki, Yoshitsugu
    Takeda, Shin'ichi
    Nagaraju, Kanneboyina
    Mouly, Vincent
    Maruyama, Rika
    Duddy, William
    Yokota, Toshifumi
    MOLECULAR THERAPY, 2017, 25 (11) : 2561 - 2572
  • [24] Optimization of therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy
    Aartsma-Rus, Annemieke
    Heemskerk, Hans
    De Winter, Christa
    Janson, Anneke
    Van Ommen, Gert-Jan
    Van Deutekom, Judith
    HUMAN GENE THERAPY, 2007, 18 (10) : 983 - 984
  • [25] Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy
    Heemskerk, Hans
    de Winter, Christa L.
    van Ommen, Gert-Jan B.
    van Deutekom, Judith C. T.
    Aartsma-Rus, Annemieke
    OLIGONUCLEOTIDE THERAPEUTICS, 2009, 1175 : 71 - 79
  • [26] Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy
    Kendall, Genevieve C.
    Mokhonova, Ekaterina I.
    Moran, Miriana
    Sejbuk, Natalia E.
    Wang, Derek W.
    Silva, Oscar
    Wang, Richard T.
    Martinez, Leonel
    Lu, Qi L.
    Damoiseaux, Robert
    Spencer, Melissa J.
    Nelson, Stanley F.
    Miceli, M. Carrie
    SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (164)
  • [27] Antisense oligonucleotide design for therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy
    Aartsma-Rus, A.
    de Winter, C. L.
    Kaman, W. E.
    Janson, A. A. M.
    van Deutekom, J. C. T.
    NEUROMUSCULAR DISORDERS, 2006, 16 (9-10) : 686 - 686
  • [28] In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping
    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
    JOURNAL OF GENE MEDICINE, 2009, 11 (03): : 257 - 266
  • [29] Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
    Engelbeen, Sarah
    O'Reilly, Daniel
    van de Vijver, Davy
    Verhaart, Ingrid
    van Putten, Maaike
    Hariharan, Vignesh
    Hassler, Matthew
    Khvorova, Anastasia
    Damha, Masad J.
    Aartsma-Rus, Annemieke
    NUCLEIC ACID THERAPEUTICS, 2023, 33 (06) : 348 - 360
  • [30] Hypoventilation in the Mdx Mouse Model of Duchenne Muscular Dystrophy
    Burns, David P.
    Arijit, Roy
    McDonald, Fiona
    Wilson, Richard J.
    O'Halloran, Ken D.
    FASEB JOURNAL, 2016, 30
12345