小柯机器人

以色列魏兹曼科学研究所Eran Hornstein等研究人员合作发现，循环miR-181是肌萎缩性侧索硬化症的预后生物标志物。2021年10月28日，国际知名学术期刊《自然—神经科学》在线发表了这一成果。

研究人员在252名具有详细临床表型的患者中调查了无血浆细胞的小RNA（miRNA）作为肌萎缩性脊髓侧索硬化症（ALS）预后生物标志物的潜力。首先，研究人员在一个纵向队列中确定了其血浆水平在疾病过程中保持稳定的miRNA。接下来，结果表明，在独立的发现和重复队列中（分别为126和122名患者），高水平的miR-181（一种富含在神经元中的miRNA）预测了大于2倍的死亡风险。

miR-181的表现与神经丝轻链（NfL）相似，当结合在一起时，miR-181+NfL建立了一个新的RNA-蛋白质生物标志物组合，并具有卓越的预后能力。因此，单独的血浆miR-181和基于miR-181+NfL的新型miRNA-蛋白质生物标志物方法提高了病人分层的精确性。基于miR-181的ALS生物标志物需要额外的验证，并有望提高临床试验的能力。

据悉，ALS是一种人类运动神经元系统的神经退行性疾病，其进展速度的变化限制了临床试验的效果。因此，更好的预后将促进治疗的进展。

附：英文原文

Title: Circulating miR-181 is a prognostic biomarker for amyotrophic lateral sclerosis

Author: Magen, Iddo, Yacovzada, Nancy Sarah, Yanowski, Eran, Coenen-Stass, Anna, Grosskreutz, Julian, Lu, Ching-Hua, Greensmith, Linda, Malaspina, Andrea, Fratta, Pietro, Hornstein, Eran

Issue&Volume: 2021-10-28

Abstract: Amyotrophic lateral sclerosis (ALS) is a relentless neurodegenerative disease of the human motor neuron system, where variability in progression rate limits clinical trial efficacy. Therefore, better prognostication will facilitate therapeutic progress. In this study, we investigated the potential of plasma cell-free microRNAs (miRNAs) as ALS prognostication biomarkers in 252 patients with detailed clinical phenotyping. First, we identified, in a longitudinal cohort, miRNAs whose plasma levels remain stable over the course of disease. Next, we showed that high levels of miR-181, a miRNA enriched in neurons, predicts agreater than two-fold risk of death in independent discovery and replication cohorts (126 and 122 patients, respectively). miR-181 performance is similar to neurofilament light chain (NfL), and when combined together, miR-181 + NfL establish a novel RNA–protein biomarker pair with superior prognostication capacity. Therefore, plasma miR-181 alone and a novel miRNA–protein biomarker approach, based on miR-181 + NfL, boost precision of patient stratification. miR-181-based ALS biomarkers encourage additional validation and might enhance the power of clinical trials.

DOI: 10.1038/s41593-021-00936-z

Source: https://www.nature.com/articles/s41593-021-00936-z

Nature Neuroscience：《自然—神经科学》，创刊于1998年。隶属于施普林格·自然出版集团，最新IF：28.771
官方网址：https://www.nature.com/neuro/
投稿链接：https://mts-nn.nature.com/cgi-bin/main.plex