enOsCas12f1-mediated exon skipping for Duchenne muscular dystrophy therapy in humanized mouse model

被引：0

作者：

Jin, Ming ^{[1
,2
]}

Lin, Jiajia ^{[1
,2
]}

Zhang, Yu ^{[3
]}

Xiao, Qingquan ^{[3
]}

Kong, Xiangfeng ^{[3
]}

Zhang, Xiumei ^{[3
]}

Shao, Zhurui ^{[3
]}

Wang, Yin ^{[3
]}

Yu, Yuyang ^{[3
]}

Li, Jinjing ^{[1
,2
]}

Chen, Wan-Jin ^{[1
,2
]}

Li, Guoling ^{[3
]}

Yang, Hui ^{[3
,4
]}

Wang, Ning ^{[1
,2
]}

机构：

[1] Fujian Med Univ, Inst Neurosci, Dept Neurol, Fujian Key Lab Mol Neurol, Fuzhou 350004, Fujian, Peoples R China

[2] Fujian Med Univ, Affiliated Hosp 1, Inst Neurosci, Fujian Key Lab Mol Neurol,Inst Neurol, Fuzhou 350004, Fujian, Peoples R China

[3] HUIDAGENE Therapeut Co Ltd, Shanghai 200131, Peoples R China

[4] Chinese Acad Sci, Shanghai Inst Biol Sci, State Key Lab Neurosci,Key Lab Primate Neurobiol,C, Inst Neurosci, Shanghai 200031, Peoples R China

来源：

JOURNAL OF GENETICS AND GENOMICS | 2024年 / 51卷 / 02期

基金：

中国国家自然科学基金;

关键词：

D O I：

10.1016/j.jgg.2023.12.003

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

引用

页码：256 / 259

页数：4

共 50 条

[31] Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model
Lin, Jiajia
Jin, Ming
Yang, Dong
Li, Zhifang
Zhang, Yu
Xiao, Qingquan
Wang, Yin
Yu, Yuyang
Zhang, Xiumei
Shao, Zhurui
Shi, Linyu
Zhang, Shu
Chen, Wan-jin
Wang, Ning
Wu, Shiwen
Yang, Hui
Xu, Chunlong
Li, Guoling
NATURE COMMUNICATIONS, 2024, 15 (01)
[32] 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
[33] 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
[34] 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
[35] 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
[36] Beyond clinical trials for antisense-mediated exon skipping for Duchenne muscular dystrophy
Aartsma-Rus, Annemieke
HUMAN GENE THERAPY, 2014, 25 (11) : A13 - A13
[37] Exon skipping therapy for Duchenne muscular dystrophy - it takes more than an antisense oligonucleotide
Aartsma-Rus, Annemieke
ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2018, 256
[38] 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
[39] Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach
Patterson, Grant
Conner, Haley
Groneman, Mecham
Blavo, Cyril
Parmar, Mayur S.
EUROPEAN JOURNAL OF PHARMACOLOGY, 2023, 947
[40] Enhanced exon skipping and Dystrophin production in a mouse model of Duchenne muscular dystrophy with EEV-PMO treatment
Kreher, N.
Li, X.
Kheirabadi, M.
Kamer, K.
Dougherty, P.
Lian, W.
Waters, C.
Gao, N.
Peddigari, S.
Stadheim, A.
Dhanabal, M.
Sethuraman, N.
Qian, Z.
NEUROMUSCULAR DISORDERS, 2021, 31 : S74 - S74

← 1 2 3 4 5 →