MicroRNA Metabolism and Dysregulation in Amyotrophic Lateral Sclerosis

Paola Rinchetti, Mafalda Rizzuti, Irene Faravelli, Stefania Corti

Research output: Contribution to journalArticlepeer-review


MicroRNAs (miRNAs) are a subset of endogenous, small, non-coding RNA molecules involved in the post-transcriptional regulation of eukaryotic gene expression. Dysregulation in miRNA-related pathways in the central nervous system (CNS) is associated with severe neuronal injury and cell death, which can lead to the development of neurodegenerative disorders, such as amyotrophic lateral sclerosis (ALS). ALS is a fatal adult onset disease characterized by the selective loss of upper and lower motor neurons. While the pathogenesis of ALS is still largely unknown, familial ALS forms linked to TAR DNA-binding protein 43 (TDP-43) and fused in sarcoma (FUS) gene mutations, as well as sporadic forms, display changes in several steps of RNA metabolism, including miRNA processing. Here, we review the current knowledge about miRNA metabolism and biological functions and their crucial role in ALS pathogenesis with an in-depth analysis on different pathways. A more precise understanding of miRNA involvement in ALS could be useful not only to elucidate their role in the disease etiopathogenesis but also to investigate their potential as disease biomarkers and novel therapeutic targets.

Original languageEnglish
Pages (from-to)2617-2630
Number of pages14
JournalMolecular Neurobiology
Issue number3
Publication statusPublished - 2018


  • ALS
  • Amyotrophic lateral sclerosis
  • Central nervous system
  • CNS
  • microRNA
  • miRNA

ASJC Scopus subject areas

  • Cellular and Molecular Neuroscience


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