Scientific rationale for the development of gene therapy strategies for Parkinson's disease.


The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has led to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the scientific rationale behind the currently tested gene therapy approaches for Parkinson's disease in some detail and pinpoint their individual unique strengths and weaknesses.

DOI: 10.1016/j.bbadis.2009.02.009
Citations per Year

287 Citations

Semantic Scholar estimates that this publication has 287 citations based on the available data.

See our FAQ for additional information.

Cite this paper

@article{Bjrklund2009ScientificRF, title={Scientific rationale for the development of gene therapy strategies for Parkinson's disease.}, author={Tomas Bj{\"{o}rklund and Deniz Kirik}, journal={Biochimica et biophysica acta}, year={2009}, volume={1792 7}, pages={703-13} }