Gentile Giulia, Morello Giovanna, La Cognata Valentina, Guarnaccia Maria, Conforti Francesca Luisa, Cavallaro Sebastiano
Institute for Biomedical Research and Innovation, Department of Biomedical Sciences, National Research Council (CNR), Via Paolo Gaifami, 18, 95126 Catania, Italy.
Medical Genetics Laboratory, Department of Pharmacy, Health and Nutritional Sciences, University of Calabria, Via Pietro Bucci, Arcavacata, 87036 Rende, Italy.
J Pers Med. 2022 May 10;12(5):770. doi: 10.3390/jpm12050770.
Alzheimer's disease (AD), Parkinson's disease (PD), and Amyotrophic Lateral Sclerosis (ALS) are representative neurodegenerative diseases (NDs) characterized by degeneration of selective neurons, as well as the lack of effective biomarkers and therapeutic treatments. In the last decade, microRNAs (miRNAs) have gained considerable interest in diagnostics and therapy of NDs, owing to their aberrant expression and their ability to target multiple molecules and pathways. Here, we provide an overview of dysregulated miRNAs in fluids (blood or cerebrospinal fluid) and nervous tissue of AD, PD, and ALS patients. By emphasizing those that are commonly dysregulated in these NDs, we highlight their potential role as biomarkers or therapeutical targets and describe the use of antisense oligonucleotides as miRNA therapies.
阿尔茨海默病(AD)、帕金森病(PD)和肌萎缩侧索硬化症(ALS)是典型的神经退行性疾病(NDs),其特征为选择性神经元变性,且缺乏有效的生物标志物和治疗方法。在过去十年中,微小RNA(miRNA)因其异常表达以及靶向多种分子和通路的能力,在神经退行性疾病的诊断和治疗方面引起了广泛关注。在此,我们概述了AD、PD和ALS患者的体液(血液或脑脊液)和神经组织中失调的miRNA。通过强调这些神经退行性疾病中常见的失调miRNA,我们突出了它们作为生物标志物或治疗靶点的潜在作用,并描述了将反义寡核苷酸用作miRNA疗法的情况。