Gene therapy rescues newborns with spinal muscular atrophy

被引:7
|
作者
Sheridan, Cormac
机构
关键词
D O I
10.1038/nbt0818-669
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:669 / 670
页数:2
相关论文
共 50 条
  • [31] Genetic therapy for spinal muscular atrophy
    Alex MacKenzie
    Nature Biotechnology, 2010, 28 : 235 - 237
  • [32] Advances in the Therapy of Spinal Muscular Atrophy
    Klotz, Jenna
    Rocha, Carolina Tesi
    Young, Sally Dunaway
    Duong, Tina
    Buu, MyMy
    Sampson, Jacinda
    Day, John W.
    JOURNAL OF PEDIATRICS, 2021, 236 : 13 - +
  • [33] Gene replacement therapy in spinal muscular atrophy: filling the data gaps
    Hagenacker, Tim
    Schara-Schmidt, Ulrike
    LANCET REGIONAL HEALTH-EUROPE, 2024, 37
  • [34] Alternative gene therapy target identified in spinal muscular atrophy mice
    Lemprière S.
    Nature Reviews Neurology, 2019, 15 (10) : 558 - 558
  • [35] Zolgensma - One-Time Gene Therapy for Spinal Muscular Atrophy
    不详
    MEDICAL LETTER ON DRUGS AND THERAPEUTICS, 2019, 61 (1577): : 113 - 114
  • [36] From gene to therapy in spinal and bulbar muscular atrophy: Are we there yet?
    Pennuto, Maria
    Rinaldi, Carlo
    MOLECULAR AND CELLULAR ENDOCRINOLOGY, 2018, 465 (0C) : 113 - 121
  • [37] A Novel AAV-Based Gene Therapy for Spinal Muscular Atrophy
    Wu, Zhenhua
    Zhu, Peixin
    Wang, Qingzeng
    Li, Zhongwan
    Chen, Shuyuan
    Dai, Li
    Li, Junhui
    Wang, Lijun
    Ye, Guojie
    MOLECULAR THERAPY, 2022, 30 (04) : 257 - 257
  • [38] Next-Generation of AAV Gene Therapy for Spinal Muscular Atrophy
    Song, Chunjuan
    Wang, Qingzeng
    Lu, Qian
    Li, Zhongwan
    Lin, Zhucheng
    Zhou, Ying
    Chen, Shuyuan
    Wang, Lijun
    Ye, Guo-Jie
    Wu, Zhenhua
    MOLECULAR THERAPY, 2023, 31 (04) : 491 - 491
  • [39] Update on gene and stem cell therapy approaches for spinal muscular atrophy
    Donnelly, Eleanor M.
    Boulis, Nicholas M.
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2012, 12 (11) : 1463 - 1471
  • [40] Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy
    Stevens, Debra
    Claborn, Melanie K.
    Gildon, Brooke L.
    Kessler, Tiffany L.
    Walker, Cheri
    ANNALS OF PHARMACOTHERAPY, 2020, 54 (10) : 1001 - 1009