Current Treatment Methods for Charcot-Marie-Tooth Diseases

被引:0
作者
Dong, Hongxian [1 ,2 ]
Qin, Boquan [1 ,2 ]
Zhang, Hui [1 ,2 ]
Lei, Lei [3 ,4 ]
Wu, Shizhou [1 ,2 ]
机构
[1] Sichuan Univ, West China Hosp, Dept Orthoped Surg, Chengdu 610041, Peoples R China
[2] Sichuan Univ, West China Hosp, Orthoped Res Inst, Chengdu 610041, Peoples R China
[3] Sichuan Univ, West China Hosp Stomatol, State Key Lab Oral Dis, Chengdu 610041, Peoples R China
[4] Sichuan Univ, West China Hosp Stomatol, Natl Clin Res Ctr Oral Dis, Chengdu 610041, Peoples R China
基金
中国国家自然科学基金;
关键词
Charcot-Marie-Tooth; pathomechanism; rehabilitation; surgery; drug therapy; gene therapy; TRIPLEX-FORMING OLIGONUCLEOTIDES; SCHWANN-CELLS; GENE-THERAPY; CYCLASE ACTIVITY; STEM-CELLS; NEUROPATHY; NEUREGULIN-1; MUTATIONS; MODELS; REHABILITATION;
D O I
10.3390/biom14091138
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Charcot-Marie-Tooth (CMT) disease, the most common inherited neuromuscular disorder, exhibits a wide phenotypic range, genetic heterogeneity, and a variable disease course. The diverse molecular genetic mechanisms of CMT were discovered over the past three decades with the development of molecular biology and gene sequencing technologies. These methods have brought new options for CMT reclassification and led to an exciting era of treatment target discovery for this incurable disease. Currently, there are no approved disease management methods that can fully cure patients with CMT, and rehabilitation, orthotics, and surgery are the only available treatments to ameliorate symptoms. Considerable research attention has been given to disease-modifying therapies, including gene silencing, gene addition, and gene editing, but most treatments that reach clinical trials are drug treatments, while currently, only gene therapies for CMT2S have reached the clinical trial stage. In this review, we highlight the pathogenic mechanisms and therapeutic investigations of different subtypes of CMT, and promising therapeutic approaches are also discussed.
引用
收藏
页数:19
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