Targeting caspase-3 as dual therapeutic benefits by RNAi facilitating brain-targeted nanoparticles in a rat model of Parkinson's disease

PLoS One. 2013 May 13;8(5):e62905. doi: 10.1371/journal.pone.0062905. Print 2013.

Abstract

The activation of caspase-3 is an important hallmark in Parkinson's disease. It could induce neuron death by apoptosis and microglia activation by inflammation. As a result, inhibition the activation of caspase-3 would exert synergistic dual effect in brain in order to prevent the progress of Parkinson's disease. Silencing caspase-3 genes by RNA interference could inhibit the activation of caspase-3. We developed a brain-targeted gene delivery system based on non-viral gene vector, dendrigraft poly-L-lysines. A rabies virus glycoprotein peptide with 29 amino-acid linked to dendrigraft poly-L-lysines could render gene vectors the ability to get across the blood brain barrier by specific receptor mediated transcytosis. The resultant brain-targeted vector was complexed with caspase-3 short hairpin RNA coding plasmid DNA, yielding nanoparticles. In vivo imaging analysis indicated the targeted nanoparticles could accumulate in brain more efficiently than non-targeted ones. A multiple dosing regimen by weekly intravenous administration of the nanoparticles could reduce activated casapse-3 levels, significantly improve locomotor activity and rescue dopaminergic neuronal loss and in Parkinson's disease rats' brain. These results indicated the rabies virus glycoprotein peptide modified brain-targeted nanoparticles were promising gene delivery system for RNA interference to achieve anti-apoptotic and anti-inflammation synergistic therapeutic effects by down-regulation the expression and activation of caspase-3.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • Blood-Brain Barrier / metabolism
  • Brain / enzymology*
  • Brain / physiopathology
  • Caspase 3 / genetics*
  • Caspase 3 / metabolism
  • Disease Models, Animal
  • Down-Regulation
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • Glycoproteins / chemistry
  • Glycoproteins / genetics
  • Male
  • Motor Activity / physiology
  • Nanoparticles / administration & dosage
  • Nanoparticles / therapeutic use
  • Parkinson Disease / enzymology
  • Parkinson Disease / physiopathology
  • Parkinson Disease / therapy*
  • Permeability
  • Polylysine / chemistry
  • Polylysine / genetics
  • RNA Interference
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics
  • Rabies virus / chemistry
  • Rats
  • Viral Proteins / chemistry
  • Viral Proteins / genetics

Substances

  • Glycoproteins
  • RNA, Small Interfering
  • Viral Proteins
  • Polylysine
  • Caspase 3

Grants and funding

This work was supported by the grant from National Natural Foundation of China (30973652), Program for New Century Excellent Talents in University and Funding for doctoral discipline in high education university (20110071110048). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.