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
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
8
引用
收藏
页码:5 / 5
页数:1
相关论文
共 50 条
  • [21] Re-engineering dystrophin for gene therapy of Duchenne muscular dystrophy
    van Raamsdonk, J
    CLINICAL GENETICS, 2002, 62 (01) : 25 - 27
  • [22] Immune responses to dystrophin: implications for gene therapy of Duchenne muscular dystrophy
    A Ferrer
    K E Wells
    D J Wells
    Gene Therapy, 2000, 7 : 1439 - 1446
  • [23] Full-length dystrophin gene therapy for Duchenne muscular dystrophy
    Duan, Dongsheng
    MOLECULAR THERAPY, 2024, 32 (09) : 2817 - 2818
  • [24] The future of Duchenne muscular dystrophy gene therapy:: Shrinking the dystrophin gene
    Roberts, ML
    Dickson, G
    CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2002, 4 (04) : 343 - 348
  • [25] Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
    Harper, SQ
    Hauser, MA
    DelloRusso, C
    Duan, DS
    Crawford, RW
    Phelps, SF
    Harper, HA
    Robinson, AS
    Engelhardt, JF
    Brooks, SV
    Chamberlain, JS
    NATURE MEDICINE, 2002, 8 (03) : 253 - 261
  • [26] Immune responses to dystrophin: implications for gene therapy of Duchenne muscular dystrophy
    Ferrer, A
    Wells, KE
    Wells, DJ
    GENE THERAPY, 2000, 7 (17) : 1439 - 1446
  • [27] Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy?
    Gorecki, Dariusz C.
    Kalinski, Pawel
    Pomeroy, Joanna
    GENE THERAPY, 2025,
  • [28] Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy
    Boennemann, Carsten G.
    Belluscio, Beth A.
    Braun, Serge
    Morris, Carl
    Singh, Teji
    Muntoni, Francesco
    NEW ENGLAND JOURNAL OF MEDICINE, 2023, 388 (24): : 2294 - 2296
  • [29] Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
    Scott Q. Harper
    Michael A. Hauser
    Christiana DelloRusso
    Dongsheng Duan
    Robert W. Crawford
    Stephanie F. Phelps
    Hollie A. Harper
    Ann S. Robinson
    John F. Engelhardt
    Susan V. Brooks
    Jeffrey S. Chamberlain
    Nature Medicine, 2002, 8 : 253 - 261
  • [30] Micro-dystrophin gene delivery for Duchenne Muscular Dystrophy: a double-blind, randomized placebo-controlled trial
    Mendell, J.
    Sheih, P.
    Sahenk, Z.
    Lehman, K.
    Lowes, L.
    Miller, N.
    Iammarino, M.
    Alfano, L.
    Woods, J.
    Skura, C.
    Mao, H.
    Staudt, L.
    Potter, R.
    Griffin, D.
    Lewis, S.
    Hu, L.
    Upadhyay, S.
    Singh, T.
    Rodino-Klapac, L.
    EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 289 - 289