CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy

被引：2
|
作者：
Wilton-Clark, Harry [1 ]
Yokota, Toshifumi [1 ]
机构：
[1] Univ Alberta, Dept Med Genet, Edmonton, AB, Canada
来源：
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT | 2023年 / 30卷
关键词：
D O I：
10.1016/j.omtm.2023.08.013
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
引用
收藏
页码：500 / 501
页数：2
相关论文
共 50 条
  • [31] CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy
    Cedric Happi Mbakam
    Gabriel Lamothe
    Guillaume Tremblay
    Jacques P. Tremblay
    Neurotherapeutics, 2022, 19 : 931 - 941
  • [32] CRISPR Base Editing Approach to Treat Duchenne Muscular Dystrophy by Skipping DMD Exon 45
    Swami, Devyani
    Gapinske, Michael
    Winter, Jackson S.
    Gapinske, Lauren
    Woods, Wendy S.
    Shirguppe, Shraddha
    Miskalis, Angelo
    Busza, Anna
    Joulani, Dana
    Kao, Colin
    Kostan, Kurt A.
    Bashir, Rashid
    Perez-Pinera, Pablo
    MOLECULAR THERAPY, 2023, 31 (04) : 609 - 609
  • [33] A Generic Strategy for CRISPR-Cas9-mediated gene tagging
    Lackner, Daniel H.
    Carre, Alexia
    Guzzardo, Paloma M.
    Banning, Carina
    Mangena, Ramu
    Henley, Tom
    Oberndorfer, Sarah
    Gapp, Bianca V.
    Nijman, Sebastian M. B.
    Brummelkamp, Thijn R.
    Burckstummer, Tilmann
    NATURE COMMUNICATIONS, 2015, 6
  • [34] A generic strategy for CRISPR-Cas9-mediated gene tagging
    Daniel H. Lackner
    Alexia Carré
    Paloma M. Guzzardo
    Carina Banning
    Ramu Mangena
    Tom Henley
    Sarah Oberndorfer
    Bianca V. Gapp
    Sebastian M.B. Nijman
    Thijn R. Brummelkamp
    Tilmann Bürckstümmer
    Nature Communications, 6
  • [35] Nanoparticle Delivery of Antisense Oligonucleotides and Their Application in the Exon Skipping Strategy for Duchenne Muscular Dystrophy
    Falzarano, Maria Sofia
    Passarelli, Chiara
    Ferlini, Alessandra
    NUCLEIC ACID THERAPEUTICS, 2014, 24 (01) : 87 - 100
  • [36] Less is more: therapeutic exon skipping for Duchenne muscular dystrophy
    Aartsma-Rus, Annemieke
    van Ommen, Gert-Jan B.
    LANCET NEUROLOGY, 2009, 8 (10): : 873 - 875
  • [37] Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges
    Arechavala-Gomeza, Virginia
    Anthony, Karen
    Morgan, Jennifer
    Muntoni, Francesco
    CURRENT GENE THERAPY, 2012, 12 (03) : 152 - 160
  • [38] New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy
    Aoki, Yoshitsugu
    Nagata, Tetsuya
    Takeda, Shin'ichi
    JOURNAL OF ADVANCED COMPUTATIONAL INTELLIGENCE AND INTELLIGENT INFORMATICS, 2012, 16 (04) : 521 - 526
  • [39] 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
  • [40] 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
12345