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Development of Gene Therapy Strategies for Neurological Diseases


The aim of the proposed project is to develop procedures for gene and cell therapy for application in the treatment of neurological diseases, in particular Parkinson's disease and epilepsy.

Approaches will involve gene therapy strategies using both ex vivo and in vivo direct injection to induce production of neurotransmitter and neurotrophic molecules.

The project includes all levels of analysis and stages of development from the production of viral vectors, genetic manipulation and encapsulation of cells, in vivo gene transfer, to the preclinical assessment of procedures in preparation for clinical trials with patients. The proposed research addresses two neurological diseases: Parkinson' s disease (PD) and epilepsy, affections chosen because their pathophysiologies suggest possible therapeutic agents: neurotransmitter, trophic factors or both. Both ex vivo and in vivo gene therapy strategies will be developed in five partially overlapping phases:

Types of cells suitable for genetic manipulation and intracerebral transplantation will be identified, and the tools to transfer genes for neurotransmitter-synthesising enzymes and neurotrophic factors constructed.
Suitable types of cells intracerebrawill be identified and the tools to transfer genes for neurotransmitter-synthesising enzymes and neurotrophic factors constructed.
Encapsulation techniques for the transplantation of genetically engineered allo and xenogeneic cell lines will be tested.
The functional properties of the transduced cells in vitro and of recombinant adenoviral constructions will be analysed in detail, and the functional efficacy of DOPA/dopamine and neurotrophic factor-producing cells in well characterised in vivo models assessed.
Alternative gene constructs where the transgene is under the control of endogenous, cell-specific promoters containing suitable regulatory elements will be studied. The aim is to obtain improved regulation and stable long-term expression of the transgenes in vivo. This will allow function of the transduced cells to be evaluated over longer time periods and in complex functional tests .
The various methods developed within the programme will be validated in small animal models and a primate model of PD.

Funding Scheme

CSC - Cost-sharing contracts


Centre National de la Recherche Scientifique
83,Boulevard De L'hopital
75013 Paris

Participants (3)

3,Pavillon 4
1011 Lausanne
Commissariat à l'Energie Atomique
4,Place Général Leclerc
91401 Orsay
Lunds Universitet
223 62 Lund