Parkin protects against neurotoxicity in the 6-hydroxydopamine rat model for Parkinson's disease

Mol Ther. 2006 Nov;14(5):716-23. doi: 10.1016/j.ymthe.2006.06.009. Epub 2006 Aug 17.

Abstract

Loss-of-function mutations in the PARK2 gene are the major cause of early onset familial Parkinson's disease. The gene product, parkin, is an E3 ligase of the ubiquitin-proteasome pathway involved in protein degradation. Dopaminergic neuron loss may result from the toxic accumulation of parkin substrates, suggesting a key role for parkin in dopaminergic neuron survival. In this study, we have investigated the neuroprotective capacity of parkin in the 6-OHDA rat model for Parkinson's disease. 6-OHDA induces the generation of reactive oxygen species leading to the degeneration of catecholaminergic neurons, but may also impair proteasome activity. Lentiviral vectors encoding human wild-type parkin or green fluorescent protein were stereotactically injected into the substantia nigra 2 weeks prior to a striatal 6-OHDA lesion. Histological analysis 1 and 3 weeks after lesioning showed a significant preservation of dopaminergic cell bodies and nerve terminals. Moreover, lesioned rats overexpressing parkin displayed a corresponding behavioral improvement as measured by the amphetamine-induced rotation test and the cylinder test. The improved performance in the amphetamine-induced rotation test lasted until 20 weeks after lesioning. Our results demonstrate that parkin acts as a potent neuroprotective agent in vivo against 6-OHDA toxic insults. These data support the therapeutic potential of parkin for the treatment of not only familial but also sporadic Parkinson's disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • Brain / drug effects
  • Brain / metabolism
  • Brain / pathology
  • Cell Line
  • Disease Models, Animal*
  • Gene Expression Regulation
  • Genetic Therapy
  • Genetic Vectors / genetics
  • Humans
  • Lentivirus / genetics
  • Motor Activity / drug effects
  • Oxidopamine / antagonists & inhibitors*
  • Oxidopamine / toxicity*
  • Parkinson Disease / metabolism*
  • Parkinson Disease / pathology*
  • Rats
  • Ubiquitin-Protein Ligases / genetics
  • Ubiquitin-Protein Ligases / metabolism*

Substances

  • Oxidopamine
  • Ubiquitin-Protein Ligases
  • parkin protein