Preclinical development of gene therapy for Parkinson’s disease.

1. Exp Neurol. 2008 Jan;209(1):72-81. Epub 2007 Aug 22.

Preclinical development of gene therapy for Parkinson’s disease.

Porras G(1), Bezard E.

Author information:
(1)CNRS UMR 5227, Universite Victor Segalen-Bordeaux 2, 33076, Bordeaux, France.

Multiple targets and pathways may be amenable to the development of gene therapy
approaches for Parkinson’s disease. This article discusses some of the cellular
and brain circuit pathways relevant to Parkinson’s disease that would be
clinically amenable to gene therapy. Approaches could be classified according to
two main categories, i.e. symptomatic vs. neuroprotective/neurorestorative
strategies. Examples of the different possibilities currently in development are
given and feature both dopaminergic and non-dopaminergic symptomatic treatments
of parkinsonian symptoms and/or L-DOPA-induced side effects, anti-apoptotic
neuroprotective strategies and growth-factor delivery for
neuroprotection/neurorestoration. While gene therapy has been mostly used so far
for enhancing the expression of the target gene, the use of dominant negative or
siRNA opens new possibilities. This, combined with the key feature of gene
delivery that offers access to intracellular signalling pathways, is likely to
further expand the number of proposed targets to be studied.

DOI: 10.1016/j.expneurol.2007.08.003
PMID: 17904121 [Indexed for MEDLINE]

Auteurs Bordeaux Neurocampus