Loss of motoneuron-specific microRNA-218 causes systemic neuromuscular failure

  • Neal D. Amin
  • , Ge Bai
  • , Jason R. Klug
  • , Dario Bonanomi
  • , Matthew T. Pankratz
  • , Wesley D. Gifford
  • , Christopher A. Hinckley
  • , Matthew J. Sternfeld
  • , Shawn P. Driscoll
  • , Bertha Dominguez
  • , Kuo Fen Lee
  • , Xin Jin
  • , Samuel L. Pfaff*
  • *Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

125 Scopus citations

Abstract

Dysfunction of microRNA (miRNA) metabolism is thought to underlie diseases affecting motoneurons. One miRNA, miR-218, is abundantly and selectively expressed by developing and mature motoneurons. Here we show that mutant mice lacking miR-218 die neonatally and exhibit neuromuscular junction defects, motoneuron hyperexcitability, and progressive motoneuron cell loss, all of which are hallmarks of motoneuron diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Gene profiling reveals that miR-218 modestly represses a cohort of hundreds of genes that are neuronally enriched but are not specific to a single neuron subpopulation. Thus, the set of messenger RNAs targeted by miR-218, designated TARGET218 , defines a neuronal gene network that is selectively tuned down in motoneurons to prevent neuromuscular failure and neurodegeneration.

Original languageEnglish
Pages (from-to)1525-1529
Number of pages5
JournalScience
Volume350
Issue number6267
DOIs
StatePublished - 18 Dec 2015
Externally publishedYes

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