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- W4206979897 abstract "Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy. PMID: 32717791 Funding information This work was supported by: Zhejiang Province Natural Science Foundation, Grant ID: LZ19H160001" @default.
- W4206979897 created "2022-01-26" @default.
- W4206979897 creator A5028619298 @default.
- W4206979897 date "2020-09-22" @default.
- W4206979897 modified "2023-09-26" @default.
- W4206979897 title "Faculty Opinions recommendation of Therapeutic strategies for duchenne muscular dystrophy: an update." @default.
- W4206979897 doi "https://doi.org/10.3410/f.738401805.793578644" @default.
- W4206979897 hasPublicationYear "2020" @default.
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