Disease-specific interventions: The use of cell and gene therapies for Parkinson disease

Approaches to repair the brain around the loss of the nigrostriatal dopaminergic pathways in Parkinson disease (PD) are not new and have been attempted over many years. However, of late, the situation has moved forward in two main ways. In the case of cell therapies, the ability to make large numbers of authentic midbrain dopaminergic neuroblasts from human pluripotent stem cell sources has turned what was an interesting avenue of research into a major area of investment and trialing, by academics in conjunction with Pharma. In the case of gene therapies, their use around dopamine replacement has waned, as the interest in using them for disease modification targeting PD-specific pathways has grown. In this chapter, we discuss all these developments and the current status of cell and gene therapies for PD.