A Novel AAV8 Vector for Microdystrophin Gene Therapy of Duchenne Muscular Dystrophy: Preclinical Studies in the Mdx Mouse

被引：0
|
作者：
Kim, SunJung [1 ]
Buss, Nicholas [1 ]
Patel, Hiren [1 ]
Yang, Lin [1 ]
Elliott, Kirk [1 ]
Qiao, Chunping [1 ]
Liu, Ye [1 ]
Fiscella, Michele [1 ]
Danos, Olivier [1 ]
机构：
[1] REGENXBIO Inc, Rockville, MD USA
来源：
MOLECULAR THERAPY | 2021年 / 29卷 / 04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：
614
引用
收藏
页码：297 / 298
页数：2
相关论文
共 50 条
  • [31] Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)
    Le Guiner, C.
    Servais, L.
    Montus, M.
    Bodvael, F.
    Gjata, B.
    Hogrel, J. Y.
    Carlier, P.
    Moullec, S.
    Masurier, C.
    Adjali, O.
    Mingozzi, F.
    Koo, T.
    Athanasopoulos, T.
    Cherel, Y.
    Mavilio, F.
    Voit, T.
    Moullier, P.
    Dickson, G.
    NEUROMUSCULAR DISORDERS, 2015, 25 : S315 - S315
  • [32] Immune Response to AAV Vector Capsid and Assessment of Eligibility for AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy
    Saha, Rima
    Meyers, Craig
    Coleman, Kirsten
    Trivedi, Prasad
    Bhake, Radhika
    Byrne, Barry J.
    Corti, Manuela
    MOLECULAR THERAPY, 2023, 31 (04) : 546 - 547
  • [33] Microdystrophin and Follistatin Combinatorial Gene Delivery To Treat a Severe Mouse Model of Duchenne Muscular Dystrophy
    Colgan, Timothy D.
    Murphy, Kate T.
    Qian, Hongwei
    Lynch, Gordon S.
    Chamberlain, Jeff S.
    Gregorevic, Paul
    MOLECULAR THERAPY, 2014, 22 : S197 - S197
  • [34] MICRODYSTROPHIN AND FOLLISTATIN COMBINATORIAL GENE DELIVERY TO TREAT A SEVERE MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY
    Colgan, T. D.
    Murphy, K. T.
    Lynch, G. S.
    Gregorevic, P.
    JOURNAL OF GENE MEDICINE, 2015, 17 (8-9): : 204 - 205
  • [35] Single-Cut Gene Editing Therapy for Duchenne Muscular Dystrophy via a Single AAV Vector
    Li, Guoling
    Lin, Jiajia
    Li, Haisen
    Jin, Ming
    Li, Zhifang
    Shi, Linyu
    Wang, Ning
    Chen, Wanjin
    Yang, Hui
    MOLECULAR THERAPY, 2023, 31 (04) : 461 - 462
  • [36] Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
    Le Guiner, Caroline
    Servais, Laurent
    Montus, Marie
    Larcher, Thibaut
    Fraysse, Bodvael
    Moullec, Sophie
    Allais, Marine
    Francois, Virginie
    Dutilleul, Maeva
    Malerba, Alberto
    Koo, Taeyoung
    Thibaut, Jean-Laurent
    Matot, Beatrice
    Devaux, Marie
    Le Duff, Johanne
    Deschamps, Jack-Yves
    Barthelemy, Ines
    Blot, Stephane
    Testault, Isabelle
    Wahbi, Karim
    Ederhy, Stephane
    Martin, Samia
    Veron, Philippe
    Georger, Christophe
    Athanasopoulos, Takis
    Masurier, Carole
    Mingozzi, Federico
    Carlier, Pierre
    Gjata, Bernard
    Hogrel, Jean-Yves
    Adjali, Oumeya
    Mavilio, Fulvio
    Voit, Thomas
    Moullier, Philippe
    Dickson, George
    NATURE COMMUNICATIONS, 2017, 8
  • [37] Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
    Caroline Le Guiner
    Laurent Servais
    Marie Montus
    Thibaut Larcher
    Bodvaël Fraysse
    Sophie Moullec
    Marine Allais
    Virginie François
    Maeva Dutilleul
    Alberto Malerba
    Taeyoung Koo
    Jean-Laurent Thibaut
    Béatrice Matot
    Marie Devaux
    Johanne Le Duff
    Jack-Yves Deschamps
    Inès Barthelemy
    Stéphane Blot
    Isabelle Testault
    Karim Wahbi
    Stéphane Ederhy
    Samia Martin
    Philippe Veron
    Christophe Georger
    Takis Athanasopoulos
    Carole Masurier
    Federico Mingozzi
    Pierre Carlier
    Bernard Gjata
    Jean-Yves Hogrel
    Oumeya Adjali
    Fulvio Mavilio
    Thomas Voit
    Philippe Moullier
    George Dickson
    Nature Communications, 8
  • [38] Effective correction of the mdx dystrophin gene mutation in the mouse model of Duchenne muscular dystrophy
    Todaro, M
    Wong, SHA
    Kita, M
    Quigley, AF
    Lowes, KN
    Marotta, R
    Cook, MJ
    Kornberg, AJ
    Kapsa, RMI
    NEUROMUSCULAR DISORDERS, 2005, 15 (9-10) : 714 - 714
  • [39] Klotho gene silencing promotes pathology in the mdx mouse model of Duchenne muscular dystrophy
    Wehling-Henricks, Michelle
    Li, Zhenzhi
    Lindsey, Catherine
    Wang, Ying
    Welc, Steven S.
    Ramos, Julian N.
    Khanlou, Negar
    Kuro-o, Makoto
    Tidball, James G.
    HUMAN MOLECULAR GENETICS, 2016, 25 (12) : 2465 - 2482
  • [40] Protein-Anchoring Therapy of Biglycan for Mdx Mouse Model of Duchenne Muscular Dystrophy
    Ito, Mikako
    Ehara, Yuka
    Li, Jin
    Inada, Kosuke
    Ohno, Kinji
    HUMAN GENE THERAPY, 2017, 28 (05) : 428 - 436
12345