Development of gene therapy for Duchenne muscular dystrophy using systemic delivery of AAV vectors

被引:1
|
作者
Chamberlain, J. S. [1 ]
Gregorevic, P. [1 ]
Schultz, B. [1 ]
Odom, G. [1 ]
Finn, E. [1 ]
机构
[1] Univ Washington, Seattle, WA 98195 USA
关键词
D O I
10.1016/S0022-510X(09)70491-5
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
引用
收藏
页码:S124 / S124
页数:1
相关论文
共 50 条
  • [1] DEVELOPMENT OF GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY USING SYSTEMIC DELIVERY OF AAV VECTORS EXPRESSING TRUNCATED DYSTROPHINS
    Chamberlain, Jeffrey S.
    Schultz, Brian
    Odom, Guy
    Wang, Zejing
    Tapscott, Stephen
    Gregorevic, Paul
    [J]. JOURNAL OF GENE MEDICINE, 2009, 11 (09): : 835 - 835
  • [2] Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
    Duan, Dongsheng
    [J]. MOLECULAR THERAPY, 2018, 26 (10) : 2337 - 2356
  • [3] Retroviral vectors for gene therapy of Duchenne muscular dystrophy
    Fassati, A
    Bresolin, N
    [J]. NEUROLOGICAL SCIENCES, 2000, 21 (05) : S925 - S927
  • [4] Retroviral vectors for gene therapy of Duchenne muscular dystrophy
    A. Fassati
    N. Bresolin
    [J]. Neurological Sciences, 2000, 21 : S925 - S927
  • [5] DEVELOPMENT OF MICRO-DYSTROPHIN VECTORS FOR GENE THERAPY OF DUCHENNE MUSCULAR DYSTROPHY
    Chamberlain, Jeffrey
    Odom, Guy
    Crudele, Julie
    Hauschka, Stephen
    [J]. MUSCLE & NERVE, 2019, 60 : S52 - S52
  • [6] Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy
    Birch, Sharla M.
    Lawlor, Michael W.
    Conlon, Thomas J.
    Guo, Lee-Jae
    Crudele, Julie M.
    Hawkins, Eleanor C.
    Nghiem, Peter P.
    Ahn, Mihye
    Meng, Hui
    Beatka, Margaret J.
    Fickau, Brittany A.
    Prieto, Juan C.
    Styner, Martin A.
    Struharik, Michael J.
    Shanks, Courtney
    Brown, Kristy J.
    Golebiowski, Diane
    Bettis, Amanda K.
    Balog-Alvarez, Cynthia J.
    Clement, Nathalie
    Coleman, Kirsten E.
    Corti, Manuela
    Pan, Xiufang
    Hauschka, Stephen D.
    Gonzalez, J. Patrick
    Morris, Carl A.
    Schneider, Joel S.
    Duan, Dongsheng
    Chamberlain, Jeffrey S.
    Byrne, Barry J.
    Kornegay, Joe. N.
    [J]. SCIENCE TRANSLATIONAL MEDICINE, 2023, 15 (677)
  • [7] Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy
    Hauser, MA
    Amalfitano, A
    KumarSingh, R
    Hauschka, SD
    Chamberlain, JS
    [J]. NEUROMUSCULAR DISORDERS, 1997, 7 (05) : 277 - 283
  • [8] COMBINATORIAL GENE THERAPY USING AAV VECTOR TECHNOLOGY TO TREAT DUCHENNE MUSCULAR DYSTROPHY
    Colgan, T. D.
    Murphy, K. T.
    Lynch, G. S.
    Gregorevic, P.
    [J]. JOURNAL OF GENE MEDICINE, 2013, 15 (8-9): : 325 - 326
  • [9] Optimised AAV-microdystrophin gene therapy for Duchenne muscular dystrophy
    Dickson, G.
    [J]. HUMAN GENE THERAPY, 2013, 24 (12) : A14 - A15
  • [10] Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
    Bowles, Dawn E.
    McPhee, Scott W. J.
    Li, Chengwen
    Gray, Steven J.
    Samulski, Jade J.
    Camp, Angelique S.
    Li, Juan
    Wang, Bing
    Monahan, Paul E.
    Rabinowitz, Joseph E.
    Grieger, Joshua C.
    Govindasamy, Lakshmanan
    Agbandje-McKenna, Mavis
    Xiao, Xiao
    Samulski, R. Jude
    [J]. MOLECULAR THERAPY, 2012, 20 (02) : 443 - 455