AAV Micro-Dystrophin Therapy Ameliorates Muscular Dystrophy in Young Adult Duchenne Muscular Dystrophy Dogs for Up to Thirty Months Following Injection

被引：0

作者：

Hakim, Chady ^{[1
]}

Kodippili, Kasun ^{[2
]}

Jenkins, Gregory ^{[2
]}

Yang, Hsiao ^{[2
]}

Pan, Xiufang ^{[2
]}

Lessa, Thais ^{[2
]}

Leach, Stacey ^{[2
]}

Emter, Craig ^{[2
]}

Yue, Yongping ^{[2
]}

Zhang, Keqing ^{[2
]}

Duan, Sean ^{[2
]}

Yao, Gang ^{[2
]}

Schneider, Joel ^{[3
]}

Yang, Nora ^{[4
]}

Chamberlain, Jeffrey ^{[5
]}

Duan, Dongsheng ^{[2
]}

机构：

[1] Univ Missouri, NCATS, Columbia, MO USA

[2] Univ Missouri, Columbia, MO USA

[3] Solid Biosci, Cambridge, MA USA

[4] NCATS, Bethesda, MD USA

[5] Univ Washington, Seatle, WA 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：

引用

页码：5 / 5

页数：1

共 50 条

[31] Optimised AAV-microdystrophin gene therapy for Duchenne muscular dystrophy
Dickson, G.
HUMAN GENE THERAPY, 2013, 24 (12) : A14 - A15
[32] Impact of myofiber centronucleation on AAV therapy efficiency for Duchenne muscular dystrophy
Mollard, A.
Peccate, C.
Julien, L.
Marais, T.
Pietri-Rouxel, F.
Benkhelifa-Ziyyat, S.
Lorain, S.
HUMAN GENE THERAPY, 2016, 27 (11) : A152 - A152
[33] Expression of normal dystrophin following myoblast transplantation to Duchenne muscular dystrophy patients
Tremblay, JP
Skuk, D
Jean-Pierre, B
Sylvain, M
Raynald, R
Marilyne, G
Brigitte, R
Chapdelaine, P
Francine, D
Lachance, JG
Deschènes, L
Senay, H
MOLECULAR THERAPY, 2004, 9 : S385 - S385
[34] Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
Le Guiner, Caroline
Xiao, Xiao
Larcher, Thibaut
Lafoux, Aude
Huchet, Corinne
Toumaniantz, Gilles
Adjali, Oumeya
Anegon, Ignacio
Remy, Syverine
Grieger, Josh
Li, Juan
Farrokhi, Vahid
Neubert, Hendrik
Owens, Jane
Mcintyre, Maritza
Moullier, Philippe
Samulski, R. Jude
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023, 30 : 30 - 47
[35] Cardiac resynchronization therapy in a young patient with Duchenne muscular dystrophy
Kono, Tamami
Ogimoto, Akiyoshi
Nishimura, Kazuhisa
Yorozuya, Toshihiro
Okura, Takafumi
Higaki, Jitsuo
INTERNATIONAL MEDICAL CASE REPORTS JOURNAL, 2015, 8 : 173 - 175
[36] New deletion constructs of dystrophin for gene therapy of duchenne muscular dystrophy (DMD)
Lochmuller, H
Orlopp, K
Dunant, P
Larochelle, N
Stucka, R
NEUROLOGY, 2000, 54 (07) : A271 - A272
[37] What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?
Wells, Dominic J.
JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY, 2019, 40 (02) : 141 - 150
[38] DYSTROPHIN PRODUCTION INDUCED BY MYOBLAST TRANSFER THERAPY IN DUCHENNE MUSCULAR-DYSTROPHY
LAW, PK
BERTORINI, TE
GOODWIN, TG
CHEN, M
FANG, QW
LI, HJ
KIRBY, DS
FLORENDO, JA
HERROD, HG
GOLDEN, GS
LANCET, 1990, 336 (8707): : 114 - 115
[39] What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?
Dominic J. Wells
Journal of Muscle Research and Cell Motility, 2019, 40 : 141 - 150
[40] Dual exon skipping in myostatin and dystrophin as a potential therapy for Duchenne muscular dystrophy
Kemaladewi, D. U.
Hoogaars, W. M. H.
van Heiningen, S. H.
van Deutekom, J. C. T.
den Dunnen, J. T.
van Ommen, G. J. B.
Aartsma-Rus, A. R.
ten Dijke, P.
't Hoen, P. A. C.
NEUROMUSCULAR DISORDERS, 2009, 19 (8-9) : 577 - 577

← 1 2 3 4 5 →