Gene therapy effective in model of infantile parkinsonism
Gene therapy effective in model of infantile parkinsonism
复制标题
基因疗法对婴儿帕金森病模型有效
DOI：
10.1038/s41582-021-00522-8
复制
发表时间：
2021
影响因子：
38.1
通讯作者：
Lemprière S
中科院分区：
文献类型：
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作者：
Lemprière S
根据一项新的研究，几种microrna （mirna）的血浆水平在1A型charcott - mary - tooth病（CMT1A）中升高，可以用作临床试验的生物标志物。Wang等人使用新一代测序和定量PCR比较了CMT1A患者和健康对照者的miRNA水平。一些肌肉相关的mirna和一组在雪旺细胞中高表达的mirna在CMT1A中增加。研究人员能够使用这些mirna中的一些来区分CMT1A和对照样本，这表明了作为疾病生物标志物的实用性。
Plasma levels of several microRNAs (miRNAs) are elevated in Charcot–Marie–Tooth disease type 1A (CMT1A) and could be used as biomarkers in clinical trials, according to new research. Wang et al. used next-generation sequencing and quantitative PCR to compare miRNA levels in individuals with CMT1A and healthy controls. Several muscle-associated miRNAs and a group of miRNAs highly expressed in Schwann cells were increased in CMT1A. The researchers were able to use some of these miRNAs to distinguish between CMT1A and control samples, suggesting utility as biomarkers of disease.