Autologous iPSC-derived dopamine neuron transplantation in a nonhuman primate Parkinson's disease model
Shuyan Wang1, Chunlin Zou2, Linlin Fu3
1Cell Therapy Center, Beijing Institute of Geriatrics, Xuanwu Hospital, Capital Medical University, and Key Laboratory of Neurodegeneration, Ministry of Education, Beijing, China; Center of Neural Injury and Repair, Beijing Institute for Brain Disorders, Beijing, China; Center of Parkinson's Disease, Beijing Institute for Brain Disorders, Beijing, China.
Abstract:
Autologous dopamine (DA) neurons are a new cell source for replacement therapy of Parkinson's disease (PD). In this study, we tested the safety and efficacy of autologous induced pluripotent stem cell (iPSC)-derived DA cells for treatment of a cynomolgus monkey PD model. Monkey bone marrow mesenchymal cells were isolated and induced to iPSCs, followed by differentiation into DA cells using a method with high efficiency. Autologous DA cells were introduced into the brain of a cynomolgus monkey PD model without immunosuppression; three PD monkeys that had received no grafts served as controls. The PD monkey that had received autologous grafts experienced behavioral improvement compared with that of controls. Histological analysis revealed no overgrowth of grafts and a significant number of surviving A9 region-specific graft-derived DA neurons. The study provided a proof-of-principle to employ iPSC-derived autologous DA cells for PD treatment using a nonhuman primate PD model.
