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Circulating Biomarkers in Muscular Dystrophies: Disease and Therapy Monitoring.
Molecular Therapy - Methods & Clinical Development ( IF 4.6 ) Pub Date : 2020-05-22 , DOI: 10.1016/j.omtm.2020.05.017
Andrie Koutsoulidou 1, 2 , Leonidas A Phylactou 1, 2
Affiliation  

Muscular dystrophies are a group of inherited disorders that primarily affect the muscle tissues. Across the muscular dystrophies, symptoms commonly compromise the quality of life in all areas of functioning. It is well noted that muscular dystrophies need reliable and measurable biomarkers that will monitor the progress of the disease and evaluate the potential therapeutic approaches. In this review, we analyze the current findings regarding the development of blood-based circulating biomarkers for different types of muscular dystrophies. We emphasize those muscular dystrophies that gained particular interest for the development of biomarkers, including Duchenne muscular dystrophy, Becker muscular dystrophy, myotonic dystrophy types 1 and 2, Ullrich congenital muscular dystrophy, congenital muscular dystrophy type 1A, Facioscapulohumeral muscular dystrophy, and limb-girdle muscular dystrophy types 2A, 2B, 2C, and 2D, recently renamed as limb-girdle muscular dystrophy R1 calpain3-related, R2 dysferlin-related, R5 γ-sarcoglycan-related, and R3 α-sarcoglycan-related. This review highlights the up-to-date progress in the development of biomarkers at the level of proteins, lipids, and metabolites, as well as microRNAs (miRNAs) that currently are the main potential biomarker candidates in muscular dystrophies.



中文翻译:

肌肉营养不良中的循环生物标志物:疾病和治疗监测。

肌营养不良是一组主要影响肌肉组织的遗传性疾病。在整个肌肉营养不良中,症状通常会损害所有功能区域的生活质量。众所周知,肌营养不良症需要可靠且可测量的生物标志物,以监测疾病的进展并评估潜在的治疗方法。在这篇综述中,我们分析了有关针对不同类型的肌肉营养不良的血液循环生物标记物发展的当前发现。我们强调那些对生物标志物的开发特别感兴趣的肌肉营养不良症,包括Duchenne肌肉营养不良,Becker肌肉营养不良,1型和2型肌强直性营养不良,Ullrich先天性肌肉营养不良,1A型先天性肌肉营养不良,面肩肱型肌营养不良和肢带型肌营养不良2A,2B,2C和2D类型最近被重新命名为肢带型肌营养不良R1 calpain3相关,R2 dysferlin相关,R5γ-sarcoglycan相关,R3α-sarcoglycanan -有关。这篇综述突出了蛋白质,脂质和代谢产物以及microRNA(miRNA)方面生物标志物开发的最新进展,这些分子目前是肌肉营养不良的主要潜在生物标志物。

更新日期:2020-06-28
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