SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy

被引:0
|
作者
Gonzalez, J. Patrick [1 ]
Brown, Kristy J. [1 ]
Lawlor, Michael W. [2 ]
Shanks, Courtney [1 ]
Schneider, Joel S. [1 ]
Morris, Carl A. [1 ]
机构
[1] Solid Biosci, Cambridge, MA USA
[2] Med Coll Wisconsin, Milwaukee, WI 53226 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
500
引用
收藏
页码:220 / 220
页数:1
相关论文
共 50 条
  • [1] Preclinical Evaluation of SGT-001 Microdystrophin Gene Transfer for Duchenne Muscular Dystrophy
    Gonzalez, J. Patrick
    Schneider, Joel S.
    Brown, Kristy J.
    Golebiowski, Diane
    Shanks, Courtney
    Ricotti, Valeria
    Laforet, Genevieve
    Quiroz, Jorge
    Morris, Carl A.
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 390 - 390
  • [2] SGT-001 Micro-dystrophin gene therapy for Duchenne muscular dystrophy
    Schneider, J.
    Gonzalez, J.
    Brown, K.
    Golebiowski, D.
    Ricotti, V.
    Quiroz, J.
    Morris, C.
    [J]. NEUROMUSCULAR DISORDERS, 2017, 27 : S188 - S188
  • [3] IGNITE-DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
    Morris, Carl A.
    Clary, Cathryn M.
    Redican, Susan
    Spector, Sidney
    Shanks, Courtney
    Brown, Kristy J.
    Schneider, Joel S.
    Gonzalez, J. Patrick
    [J]. MOLECULAR THERAPY, 2021, 29 (04) : 135 - 136
  • [4] IGNITE DMD Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: Long-Term Outcomes and Biomarker Update
    Morris, Carl A.
    Dreghici, Roxana Donisa
    Redican, Susan
    Lawrence, Jeffry
    Brown, Kristy J.
    Wang, Fei
    Gonzalez, Patrick
    Schneider, Joel S.
    Clary, Cathryn
    Shieh, Perry B.
    Byrne, Barry J.
    [J]. MOLECULAR THERAPY, 2022, 30 (04) : 553 - 554
  • [5] Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy
    Hart, Cora C.
    Lee, Young Il
    Xie, Jun
    Gao, Guangping
    Lin, Brian L.
    Hammers, David W.
    Sweeney, H. Lee
    [J]. JCI INSIGHT, 2024, 9 (11)
  • [6] Optimised AAV-microdystrophin gene therapy for Duchenne muscular dystrophy
    Dickson, G.
    [J]. HUMAN GENE THERAPY, 2013, 24 (12) : A14 - A15
  • [7] DMD patients treated by SGT-001 microdystrophin gene therapy improve in the objective endpoint of spontaneous walking velocity
    Servais, L.
    Gonzalez, P.
    Morris, C.
    Shieh, P.
    Byrne, B.
    Annoussamy, M.
    Dreghici, Donisa R.
    [J]. NEUROMUSCULAR DISORDERS, 2022, 32
  • [8] IGNITE DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy: Update on Pulmonary Outcomes
    Mayer, O.
    Byrne, B.
    Salabarria, S.
    Berthy, J.
    Corti, M.
    Redican, S.
    Lawrence, J.
    Brown, K.
    Shanks, C.
    Spector, S.
    Gonzalez, J.
    Schneider, J.
    Morris, C.
    Clary, C.
    [J]. ANNALS OF NEUROLOGY, 2021, 90 : S89 - S89
  • [9] Compartmentalized vascular delivery of microdystrophin as a gene therapy treatment for Duchenne muscular dystrophy
    Rodino-Klapac, Louise R.
    Montgomery, Chrystal L.
    Clark, K. Reed
    Coley, Brian
    Jensen, Ryan
    Chicoine, Louis G.
    Mendell, Jery
    [J]. NEUROLOGY, 2007, 68 (12) : A1 - A2
  • [10] Complementary Techniques to Evaluate Microdystrophin Expression in Duchenne Muscular Dystrophy Gene Therapy Studies
    Fickau, Brittany
    Beatka, Margaret
    Gonzales, Patrick
    Meng, Hui
    Birch, Sharla
    Morris, Carl A.
    Ayres, Samuel
    Tinklenberg, Jennifer
    Avond, Mark Vanden
    Kornegay, Joe
    Schneider, Joel S.
    Brown, Kristy J.
    Lawlor, Michael W.
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 396 - 397