Therapeutic tools for inherited neuropathies

被引:2
|
作者
Pereon, Y. [1 ]
机构
[1] CHU Nantes, Lab Explorat Fonct, FILNEMUS, Euro NMD ,Ctr Reference Malad Neuromusculaires AOC, Nantes, France
关键词
Gene therapy; Gene expression therapy; Antisense oligonucleotides; SiRNA; GENE-THERAPY; MYELIN; IMPROVE;
D O I
10.1016/j.neurol.2022.12.001
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Inherited neuropathies are a genetically and phenotypically heterogenous group of dis-orders leading to sensory and motor dysfunction. For years, these neuropathies have been considered as non-treatable diseases, as no drug is able to induce nerve regrowth. Progress in molecular tools has changed this view and several neuropathies can now be efficiently treated. Some more will be treatable in the upcoming years. Basically, these new treatments can be divided into four categories, depending on the target: gene therapy; gene expression therapy; protein modification or replacement (enzyme replacement therapy, ERT); downs-tream therapies. In this short review, we will provide a few examples for each of them in the field of peripheral neuropathies. (c) 2022 Elsevier Masson SAS. All rights reserved.
引用
收藏
页码:5 / 9
页数:5
相关论文
共 50 条
  • [1] Therapeutic strategies for the inherited neuropathies
    Shy, Michael E.
    NEUROMOLECULAR MEDICINE, 2006, 8 (1-2) : 255 - 278
  • [2] Therapeutic strategies for the inherited neuropathies
    Michael E. Shy
    NeuroMolecular Medicine, 2006, 8 : 255 - 278
  • [3] Therapeutic Approaches to Inherited Optic Neuropathies
    Yu-Wai-Man, Patrick
    SEMINARS IN NEUROLOGY, 2015, 35 (05) : 578 - 586
  • [4] Novel therapeutic approaches in inherited neuropathies: a systematic review
    Hustinx, M.
    Shorrocks, A.
    Servais, L.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S75 - S75
  • [5] Novel Therapeutic Approaches in Inherited Neuropathies: A Systematic Review
    Hustinx, Manon
    Shorrocks, Ann-Marie
    Servais, Laurent
    PHARMACEUTICS, 2023, 15 (06)
  • [6] Inherited Neuropathies
    Fridman, Vera
    Reilly, M. M.
    SEMINARS IN NEUROLOGY, 2015, 35 (04) : 407 - 423
  • [7] INHERITED NEUROPATHIES
    THOMAS, PK
    ELECTROENCEPHALOGRAPHY AND CLINICAL NEUROPHYSIOLOGY, 1985, 61 (03): : S18 - S18
  • [8] Inherited neuropathies
    Kleopa, KA
    Scherer, SS
    NEUROLOGIC CLINICS, 2002, 20 (03) : 679 - +
  • [9] The inherited neuropathies
    Klein, Christopher J.
    NEUROLOGIC CLINICS, 2007, 25 (01) : 173 - +
  • [10] Inherited Neuropathies
    Angelo Schenone
    Lucilla Nobbio
    Margherita Monti Bragadin
    Giulia Ursino
    Marina Grandis
    Current Treatment Options in Neurology, 2011, 13 : 160 - 179