Hum Gene Ther. 2016 Oct 20. [Epub ahead of print]

Stem cells for modeling and therapy of Parkinson's disease.

Zhang Q^1,2, Chen W^3,4, Tan S⁵, Lin T^3,6.

Abstract

Parkinson's disease (PD) is the second most frequent neurodegenerative disease after Alzheimer's disease, which is characterized by low level of dopamine expressing in the striatum and deteriorated dopaminergic neurons (DAn) in Substantia nigra pars compacta (SNpc). Generation of PD-derived DAn including differentiation of human embryonic stem cell (hESC), human neural stem cell (hNSC), human induced pluripotent stem cell (hiPSC) and directly reprogramming provide an ideal tool to model PD, which created the possibilities of mimicking key essential pathological processes charactering single cell changes in vitro. Furthermore, thanks to the understanding of molecular neuropathogenesis of PD and new advances in stem cell technology, optimal functionally transplanted DAn with targeted correction and transgene-free insertion is anticipated to be generated for use of cell transplantation. In this review, we will elucidate stem cell technology for modeling PD and offering desired safe cell resources for cell transplantation therapy.