Neurodegeneration as an RNA disorder

Prog Neurobiol. 2012 Dec;99(3):293-315. doi: 10.1016/j.pneurobio.2012.09.006. Epub 2012 Oct 10.


Neurodegenerative diseases constitute one of the single most important public health challenges of the coming decades, and yet we presently have only a limited understanding of the underlying genetic, cellular and molecular causes. As a result, no effective disease-modifying therapies are currently available, and no method exists to allow detection at early disease stages, and as a result diagnoses are only made decades after disease pathogenesis, by which time the majority of physical damage has already occurred. Since the sequencing of the human genome, we have come to appreciate that the transcriptional output of the human genome is extremely rich in non-protein coding RNAs (ncRNAs). This heterogeneous class of transcripts is widely expressed in the nervous system, and is likely to play many crucial roles in the development and functioning of this organ. Most exciting, evidence has recently been presented that ncRNAs play central, but hitherto unappreciated roles in neurodegenerative processes. Here, we review the diverse available evidence demonstrating involvement of ncRNAs in neurodegenerative diseases, and discuss their possible implications in the development of therapies and biomarkers for these conditions.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Expression Regulation / genetics
  • Humans
  • MicroRNAs / genetics
  • MicroRNAs / metabolism
  • Neurodegenerative Diseases / genetics*
  • Neurodegenerative Diseases / metabolism
  • Neurodegenerative Diseases / physiopathology
  • RNA, Untranslated / genetics*
  • RNA, Untranslated / physiology
  • Transcription, Genetic / genetics*
  • Trinucleotide Repeats / genetics


  • MicroRNAs
  • RNA, Untranslated