Gene Therapy for Parkinson's Disease

Advances in molecular biology and virology in recent years have enabled the technology of gene transfer to proceed forward. Parkinson's disease (PD) is a particularly appropriate target for gene therapy since the brain pathology is fully characterized and relatively well circumscribed largely within the nigrostriatal dopaminergic neurons. In addition, the search for genetic mutations responsible for familial forms of PD has accelerated in recent years with several genes or loci already identified. Mutations in the parkin gene are linked to the autosomal recessive form known as autosomal recessive juvenile parkinsonism, park2. Therefore, parkin gene therapy can be effective in PD caused by parkin gene mutations, which are inherited as an autosomal recessive trait. Intriguingly, several studies, including our reports, have suggested the possibility that parkin gene therapy could treat a subset of patients with PD who have mutations in the alpha-synuclein gene. Furthermore, if indeed parkin overexpression broadly corrects anatomical degeneration in the substantia nigra and striatum, this might be a potential therapy for alpha-synucleinopathy.