The emerging roles of microRNAs in the pathogenesis of frontotemporal dementia-amyotrophic lateral sclerosis (FTD-ALS) spectrum disorders

J Neurogenet. Mar-Jun 2014;28(1-2):30-40. doi: 10.3109/01677063.2013.876021. Epub 2014 Feb 10.


Increasing evidence suggests that frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) share some clinical, pathological, and molecular features as part of a common neurodegenerative spectrum disorder. In recent years, enormous progress has been made in identifying both pathological proteins and genetic mutations associated with FTD-ALS. However, the molecular pathogenic mechanisms of disease onset and progression remain largely unknown. Recent studies have uncovered unexpected links between FTD-ALS and multiple aspects of RNA metabolism, setting the stage for further understanding of the disorder. Here, the authors will focus on microRNAs and review the emerging roles of these small RNAs in several aspects of FTD-ALS pathogenesis.

Keywords: ALS; C9ORF72; CHMP2B; FTD; FUS; MicroRNA; TDP-43; neurodegeneration; progranulin.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / genetics*
  • Amyotrophic Lateral Sclerosis / metabolism
  • Amyotrophic Lateral Sclerosis / pathology*
  • DNA-Binding Proteins / genetics
  • DNA-Binding Proteins / metabolism
  • Frontotemporal Dementia / genetics*
  • Frontotemporal Dementia / metabolism
  • Frontotemporal Dementia / pathology*
  • Humans
  • MicroRNAs / genetics*
  • MicroRNAs / metabolism
  • Mutation / genetics


  • DNA-Binding Proteins
  • MicroRNAs

Supplementary concepts

  • Frontotemporal Dementia With Motor Neuron Disease