期刊
CELL STEM CELL
卷 16, 期 3, 页码 269-274出版社
CELL PRESS
DOI: 10.1016/j.stem.2015.01.018
关键词
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资金
- Harvard Stem Cell Institute Miller Consortium
- Udall Parkinson's Disease Center of Excellence [P50 NS39793]
- Department of Defense [WX81XWH-11-1-0069]
- Orchard Foundation
- Poul Hansen Family
- Consolidated Anti-Aging Foundation
- Harold and Ronna Cooper Family
- National Center for Research Resources [RR00168]
- Office of Research Infrastructure Programs [OD011103]
Autologous transplantation of patient-specific induced pluripotent stem cell (iPSC)-derived neurons is a potential clinical approach for treatment of neurological disease. Preclinical demonstration of long-term efficacy, feasibility, and safety of iPSC-derived dopamine neurons in non-human primate models will be an important step in clinical development of cell therapy. Here, we analyzed cynomolgus monkey (CM) iPSC-derived midbrain dopamine neurons for up to 2 years following autologous transplantation in a Parkinson's disease (PD) model. In one animal, with the most successful protocol, we found that unilateral engraftment of CM-iPSCs could provide a gradual onset of functional motor improvement contralateral to the side of dopamine neuron transplantation, and increased motor activity, without a need for immunosuppression. Postmortem analyses demonstrated robust survival of midbrain-like dopaminergic neurons and extensive outgrowth into the transplanted putamen. Our proof of concept findings support further development of autologous iPSC-derived cell transplantation for treatment of PD.
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