Duchenne muscular dystrophy: Current state and therapeutic perspectives

被引：0
作者：
Le Guen, Y. T. [1 ]
Le Gall, T. [1 ]
Laurent, V [1 ]
D'Arbonneau, F. [1 ]
Braun, S. [2 ]
Montier, T. [1 ,3 ]
机构：
[1] Univ Bretagne Occidentale, INSERM, Genet Genom Fonct & Biotechnol, EFS,UMR 1078, 22 Rue Camille Desmoulins,CS 93837, F-29238 Brest 3, France
[2] AFM Telethon, 1 Rue Int,BP59, F-91002 Evry, France
[3] CHRU Brest, Serv Genet Med & Biol Reprod, Ctr Reference Malad Rares Malad Neuromusculaires, F-29200 Brest, France
来源：
BULLETIN DE L ACADEMIE NATIONALE DE MEDECINE | 2021年 / 205卷 / 05期
关键词：
Muscular Dystrophy; Duchenne; Therapeutics; Gene Therapy; GENE-THERAPY; FEMALE CARRIERS; MUSCLE; GLUCOCORTICOIDS; CHILDREN; VECTORS; IMPACT;
D O I：
10.1016/j.banm.2020.10.019
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Duchenne muscular dystrophy is one of the most frequent muscular dystrophies, with 250,000 affected people worldwide. It results from mutations occurring in the dystrophin gene, the largest known in the human genome. Despite current therapies allow for a better clinical prognosis, the life span and the quality of life of patients still remain clearly lower than those in the general population. New therapeutic approaches are currently investigated, giving new perspectives and hopes for the care of this severe disease. (C) 2021 l'Academie nationale de medecine. Published by Elsevier Masson SAS. All rights reserved.
引用
收藏
页码：509 / 518
页数：10
相关论文
共 50 条
  • [21] Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy
    Dominic J. Wells
    [J]. Journal of Muscle Research & Cell Motility, 2006, 27 : 387 - 398
  • [22] Common therapeutic advances for Duchenne muscular dystrophy (DMD)
    Salmaninejad, Arash
    Abarghan, Yousef Jafari
    Qomi, Saeed Bozorg
    Bayat, Hadi
    Yousefi, Meysam
    Azhdari, Sara
    Talebi, Samaneh
    Mojarrad, Majid
    [J]. INTERNATIONAL JOURNAL OF NEUROSCIENCE, 2021, 131 (04) : 370 - 389
  • [23] Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy
    Wells, Dominic J.
    [J]. JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY, 2006, 27 (5-7) : 387 - 398
  • [24] Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy
    D'Ambrosio, Eleonora S.
    Mendell, Jerry R.
    [J]. NEUROTHERAPEUTICS, 2023, 20 (06) : 1669 - 1681
  • [25] Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy
    Mendell, Jerry R.
    Rodino-Klapac, Louise R.
    Malik, Vinod
    [J]. JOURNAL OF CHILD NEUROLOGY, 2010, 25 (09) : 1145 - 1148
  • [26] Molecular and Biochemical Therapeutic Strategies for Duchenne Muscular Dystrophy
    Krishna, Lakshmi
    Prashant, Akila
    Kumar, Yogish H.
    Paneyala, Shasthara
    Patil, Siddaramappa J.
    Ramachandra, Shobha Chikkavaddaragudi
    Vishwanath, Prashant
    [J]. NEUROLOGY INTERNATIONAL, 2024, 16 (04): : 731 - 760
  • [27] Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy
    Happi Mbakam, Cedric
    Lamothe, Gabriel
    Tremblay, Jacques P.
    [J]. FRONTIERS IN MEDICINE, 2022, 9
  • [28] Duchenne muscular dystrophy: disease mechanism and therapeutic strategies
    Angulski, Addeli Bez Batti
    Hosny, Nora
    Cohen, Houda
    Martin, Ashley A.
    Hahn, Dongwoo
    Bauer, Jack
    Metzger, Joseph M.
    [J]. FRONTIERS IN PHYSIOLOGY, 2023, 14
  • [29] CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives
    Chen, Guofang
    Wei, Tingyi
    Yang, Hui
    Li, Guoling
    Li, Haisen
    [J]. CELLS, 2022, 11 (19)
  • [30] Current and emerging treatment strategies for Duchenne muscular dystrophy
    Mah, Jean K.
    [J]. NEUROPSYCHIATRIC DISEASE AND TREATMENT, 2016, 12 : 1795 - 1807
12345