Induced pluripotent stem cells: a novel tool for modelling and treating Parkinson’s disease

Abstract

Parkinson's disease (PD) is a chronic, progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons primarily affecting the substantia nigra pars compacta, leading to a deficiency of dopamine in the striatum. Currently, existing treatments of PD only provide symptomatic relief and a permanent cure is yet to be discovered. Although animal models have provided valuable insights into the pathophysiology underlying PD, they are unable to recapitulate the full range of symptoms of human PD mainly due to species differences. These factors highlight an important clinical unmet need for developing cellular models of PD to study pathogenic mechanisms in-depth and identify potential drug targets. iPSCs provide a unique platform to model certain human diseases in vitro and offer the potential to develop cell-transplantation therapies as an innovative treatment strategy for PD. This comprehensive review discusses the utilization of patient-specific iPSCs to study disease mechanisms at a molecular level and discusses the challenges and potential solutions to overcome them.