{"title":"有希望的治疗杜氏肌营养不良:利用核酸疗法恢复营养不良蛋白的表达","authors":"G. Hu, Chen Chen","doi":"10.53941/ijddp.0201002","DOIUrl":null,"url":null,"abstract":"Review\nPromising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics\n\nGuo Hu and Chen Chen *\n\n\nDivision of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.\n* Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422\n \n \nReceived: 10 October 2022\nAccepted: 4 November 2022\nPublished: 11 January 2023\n \n\nAbstract: Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.","PeriodicalId":94047,"journal":{"name":"International journal of drug discovery and pharmacology","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2023-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics\",\"authors\":\"G. Hu, Chen Chen\",\"doi\":\"10.53941/ijddp.0201002\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Review\\nPromising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics\\n\\nGuo Hu and Chen Chen *\\n\\n\\nDivision of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.\\n* Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422\\n \\n \\nReceived: 10 October 2022\\nAccepted: 4 November 2022\\nPublished: 11 January 2023\\n \\n\\nAbstract: Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.\",\"PeriodicalId\":94047,\"journal\":{\"name\":\"International journal of drug discovery and pharmacology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2023-02-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"International journal of drug discovery and pharmacology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.53941/ijddp.0201002\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"International journal of drug discovery and pharmacology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.53941/ijddp.0201002","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics
Review
Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics
Guo Hu and Chen Chen *
Division of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
* Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422
Received: 10 October 2022
Accepted: 4 November 2022
Published: 11 January 2023
Abstract: Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.