ABSTRACT
Gene therapy involves the introduction of a novel genetic sequence to a specific population of cells, either ex vivo or in vivo, for the purpose of de novo synthesis of proteins or various peptides. An engineered vector containing the desired genetic sequence facilitates the transduction of the target cells. Gene therapy for Parkinson’s disease (PD) can be used to continuously deliver large molecules like dopamine (DA), its precursors, enzymes, and various trophic factors inside the blood-brain barrier (BBB) without the use of indwelling mechanical devices such as pumps or reservoirs. A neurosurgical procedure is still used to deliver the gene therapy vector to a localized delivery area. Therefore, the treatment of PD continues to be a major focus for neurological gene therapists, because it will be sufficient to deliver a transgene or reduce expression of a gene in a particular anatomical region to the exclusion of global delivery. The present chapter will review the current gene therapy strategies under investigation for the treatment of PD and will introduce gene therapies that are potentially on the clinical horizon.
