Publication Type Case Report
Authors Schweitzer J, Song B, Herrington T, Park T, Lee N, Ko S, Jeon J, Cha Y, Kim K, Li Q, Henchcliffe C, Kaplitt M, Neff C, Rapalino O, Seo H, Lee I, Kim J, Kim T, Petsko G, Ritz J, Cohen B, Kong S, Leblanc P, Carter B, Kim K
Journal N Engl J Med
Volume 382
Issue 20
Pagination 1926-1932
Date Published 05/14/2020
ISSN 1533-4406
Keywords Dopaminergic Neurons, Induced Pluripotent Stem Cells, Parkinson Disease, Pars Compacta
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 immunosuppression. Positron-emission tomography with the use of fluorine-18-L-dihydroxyphenylalanine suggested graft survival. Clinical measures of symptoms of Parkinson's disease after surgery stabilized or improved at 18 to 24 months after implantation. (Funded by the National Institutes of Health and others.).
DOI 10.1056/NEJMoa1915872
PubMed ID 32402162
PubMed Central ID PMC7288982
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