articleNew England Journal of MedicineMay 13, 2020BRONZE OA

Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease

Center for Children · Laboratory of Molecular Genetics · +9 more institutions

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Abstract

We report the implantation of patient-derived midbrain dopaminergic progenitor cells, differentiated in vitro from autologous induced pluripotent stem cells (iPSCs), in a patient with idiopathic Parkinson's disease. The patient-specific progenitor cells were produced under Good Manufacturing Practice conditions and characterized as having the phenotypic properties of substantia nigra pars compacta neurons; testing in a humanized mouse model (involving peripheral-blood mononuclear cells) indicated an absence of immunogenicity to these cells. The cells were implanted into the putamen (left hemisphere followed by right hemisphere, 6 months apart) of a patient with Parkinson's disease, without the need for…

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