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

页码：500 / 501

页数：2

共 50 条

[31] CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy [J].

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 [J].

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 [J].

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 [J].

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 [J].

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 [J].

Aartsma-Rus, Annemieke ;

van Ommen, Gert-Jan B. .

LANCET NEUROLOGY, 2009, 8 (10) :873-875

[37] New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy [J].

Aoki, Yoshitsugu ;

Nagata, Tetsuya ;

Takeda, Shin'ichi .

JOURNAL OF ADVANCED COMPUTATIONAL INTELLIGENCE AND INTELLIGENT INFORMATICS, 2012, 16 (04) :521-526

[38] Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges [J].

Arechavala-Gomeza, Virginia ;

Anthony, Karen ;

Morgan, Jennifer ;

Muntoni, Francesco .

CURRENT GENE THERAPY, 2012, 12 (03) :152-160

[39] Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations [J].

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

[40] Towards clinical studies on antisense-mediated exon skipping in Duchenne Muscular Dystrophy [J].

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

← 1 2 3 4 5 →