Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

被引：149
|
作者：
Wilson, James M. [1 ]
Flotte, Terence R. [2 ]
机构：
[1] Univ Penn, Perelman Sch Med, Gene Therapy Program, Philadelphia, PA 19104 USA
[2] Univ Massachusetts, Horae Gene Therapy Ctr, Med Sch, Worcester, MA 01605 USA
来源：
HUMAN GENE THERAPY | 2020年 / 31卷 / 13-14期
关键词：
D O I：
10.1089/hum.2020.182
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
收藏
页码：695 / 696
页数：2
相关论文
共 50 条
  • [41] ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment
    Shieh, Perry B.
    Kuntz, Nancy
    Smith, Barbara
    Dowling, James
    Mueller-Felber, Wolfgang
    Boennemann, Carsten G.
    Servais, Laurent
    Muntoni, Francesco
    Blaschek, Astrid
    Neuhaus, Sarah
    Alfano, Lindsay N.
    Beggs, Alan H.
    Buj-Bello, Ana
    Childers, Martin
    Duong, Tina
    Graham, Robert J.
    Jain, Minal
    James, Emma S.
    Lawlor, Michael W.
    Lee, Jun
    MacBean, Victoria
    Mavilio, Fulvio
    Murtagh, Michael
    Noursalehi, Mojtaba
    Prasad, Suyash
    Rico, Salvador
    NEUROLOGY, 2020, 94 (15)
  • [42] ASPIRO Gene therapy trial in X-linked myotubular myopathy (XLMTM): Update on preliminary safety and efficacy findings up to 72 weeks post-treatment
    Shieh, P.
    Kuntz, N.
    Bonnemann, C.
    Mueller-Felber, W.
    Lawlor, M. W.
    Servais, L.
    Smith, B.
    Noursalehi, M.
    Prasad, S.
    Dowling, J.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S65 - S66
  • [43] Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward
    Bennett, Jean
    MOLECULAR THERAPY, 2017, 25 (05) : 1076 - 1094
  • [44] Neuromuscular Ultrasound for Diaphragm Assessment Following Gene Replacement Therapy in a Canine Model of X-Linked Myotubular Myopathy (XLMTM)
    Sarwal, Aarti
    Serfas, J. D.
    Mitchell, Erin
    Cartwright, Michael
    Buj-Bello, Anna
    Beggs, Alan
    Childers, Martin
    NEUROLOGY, 2013, 80
  • [45] Cloning and characterization of an alternatively spliced gene in proximal Xq28 deleted in two patients with intersexual genitalia and myotubular myopathy
    Laporte, J
    Kioschis, P
    Hu, LJ
    Kretz, C
    Carlsson, B
    Poustka, A
    Mandel, JL
    Dahl, N
    GENOMICS, 1997, 41 (03) : 458 - 462
  • [46] Gene Therapy for Nonischemic Cardiomyopathy Moving Forward by Learning From Lessons of the Past
    Regnier, Michael
    Moussavi-Harami, Farid
    JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2017, 70 (14) : 1757 - 1759
  • [47] Gene Therapy Leads to Dose-Dependent Transcriptome Remodeling and Provides Biomarkers of Therapeutic Efficacy in X-Linked Myotubular Myopathy
    Dupont, Jean-Baptiste
    Guo, Jianjun
    Gray, John T.
    Buj-Bello, Ana
    Childers, Martin K.
    Mack, David L.
    MOLECULAR THERAPY, 2018, 26 (05) : 5 - 6
  • [48] Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma
    Philippidis, Alex
    HUMAN GENE THERAPY, 2022, 33 (17-18) : 842 - 844
  • [49] TWO CASES OF HYPOTHYROID MYOPATHY WITH IMPROVEMENT AFTER THYROID REPLACEMENT THERAPY
    Sampat, Radhika
    Garcia-Santibanez, Rocio
    Glass, Jonathan
    MUSCLE & NERVE, 2016, 54 (03) : 591 - 591
  • [50] Two boys die in gene therapy trial
    不详
    SCIENCE, 2020, 369 (6499) : 13 - 13
12345