Drp1 inhibition attenuates neurotoxicity and dopamine release deficits in vivo

Nat Commun. 2014 Nov 5:5:5244. doi: 10.1038/ncomms6244.

Abstract

Mitochondrial dysfunction has been reported in both familial and sporadic Parkinson's disease (PD). However, effective therapy targeting this pathway is currently inadequate. Recent studies suggest that manipulating the processes of mitochondrial fission and fusion has considerable potential for treating human diseases. To determine the therapeutic impact of targeting these pathways on PD, we used two complementary mouse models of mitochondrial impairments as seen in PD. We show here that blocking mitochondrial fission is neuroprotective in the PTEN-induced putative kinase-1 deletion (PINK1(-/-)) and 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine mouse models. Specifically, we show that inhibition of the mitochondrial fission GTPase dynamin-related protein-1 (Drp1) using gene-based and small-molecule approaches attenuates neurotoxicity and restores pre-existing striatal dopamine release deficits in these animal models. These results suggest Drp1 inhibition as a potential treatment for PD.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine
  • Animals
  • Disease Models, Animal
  • Dopamine / metabolism*
  • Dopaminergic Neurons / metabolism
  • Dynamins / antagonists & inhibitors*
  • HEK293 Cells
  • Humans
  • Male
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mitochondrial Dynamics*
  • Mitochondrial Proteins / metabolism
  • Parkinson Disease / metabolism
  • Parkinson Disease / therapy*
  • Protein Kinases / genetics

Substances

  • FIS1 protein, mouse
  • Mitochondrial Proteins
  • 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine
  • Protein Kinases
  • PTEN-induced putative kinase
  • Dnm1l protein, mouse
  • Dynamins
  • Dopamine