11. Novel Molecular Therapeutics in Parkinson's Disease

  1. David Whitehouse2 and
  2. Ralph Rapley3
  1. Susana Gonçalves,
  2. Hugo Vicente Miranda and
  3. Tiago F. Outeiro

Published Online: 16 MAR 2012

DOI: 10.1002/9781119967309.ch11

Molecular and Cellular Therapeutics

Molecular and Cellular Therapeutics

How to Cite

Gonçalves, S., Miranda, H. V. and Outeiro, T. F. (2012) Novel Molecular Therapeutics in Parkinson's Disease, in Molecular and Cellular Therapeutics (eds D. Whitehouse and R. Rapley), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/9781119967309.ch11

Editor Information

  1. 2

    School of Life Sciences, University of Hertfordshire, UK

  2. 3

    University of Hertfordshire, UK

Author Information

  1. Cell and Molecular Neuroscience Unit, Instituto de Medicina Molecular, Av. Prof. Egas Moniz, 1649-028 Lisboa, Portugal

Publication History

  1. Published Online: 16 MAR 2012
  2. Published Print: 16 FEB 2012

ISBN Information

Print ISBN: 9780470748145

Online ISBN: 9781119967309



  • Parkinson's disease;
  • novel molecular therapeutics;
  • Protein degradation systems;
  • Protein folding systems;
  • Glial cell-linederived neurotrophic factor (GDNF);
  • Adeno-associated viral vectors (AAVs);
  • Lactoferrin (Lf)


Parkinson's disease is the most common movement disorder, characterized by the loss of dopaminergic neurons from the substantia nigra and by the accumulation of intraneuronal alpha-synuclein (a-syn) inclusions called Lewy bodies. a-syn misfolding and aggregation are thought to enable aberrant protein-protein interactions and result in the formation of inclusion bodies. This culminates in the impairment of several essential cellular functions such as intracellular trafficking, protein degradation, and mitochondrial function, eventually leading to cell death.

Currently there is no cure for Parkinson's disease and the available therapies are only symptomatic. Here, we outline how some of the recent molecular and genetic research might be used as the basis for the development of novel therapeutic strategies.