Perspectives of Parkinsonâs Disease Therapies using Induced Pluripotent Stem Cells
Parkinson’s Disease (PD) is an intractable disease resulting in localized neurodegeneration of dopaminergic neurons of the substantia nigra pars compacta. Many current therapies of PD are symptomatic, but no current option for clinical-grade disease modifying treatment exists. Fortunately, recent advances in the field of cellular reprogramming now allow for previously unattainable cell therapies and modeling of PD using induced pluripotent stem cells (iPSCs) to potentially restore a disease-free state. iPSCs can be selectively differentiated into a dopaminergic neuron fate to model endogenous physiology and pathogenesis. iPSC lines can also be established with genetically-linked PD. These patient-specific cell lines are then genetically corrected in mutations of influence and can be subsequently transplanted back into the patient to reestablish function. This year, induced pluripotent stem cells iPSCs entered the first human trial for PD therapy. This form of cell therapy has shown promising results in other model organisms and is currently one of our best options in slowing or even halting the progression of PD. Here we examine the genetic contributions that have reshaped our understanding of PD, as well as the advantages and applications of iPSCs for modeling disease and clinical therapies.
Theo Stoddard Bennett and Renee Reijo Pera