Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease
- Authors
- Schweitzer, Jeffrey S.; Song, Bin; Herrington, Todd M.; Park, Tae-Yoon; Lee, Nayeon; Ko, Sanghyeok; Jeon, Jeha; Cha, Young; Kim, Kyungsang; Li, Quanzheng; Henchcliffe, Claire; Kaplitt, Michael; Neff, Carolyn; Rapalino, Otto; SEO, HYE MYUNG; Lee, In-Hee; Kim, Jisun; Kim, Taewoo; Petsko, Gregory A.; Ritz, Jerome; Cohen, Bruce M.; Kong, Sek-Won; Leblanc, Pierre; Carter, Bob S.; Kim, Kwang-Soo
- Issue Date
- May-2020
- Publisher
- Massachusetts Medical Society
- Citation
- New England Journal of Medicine, v.382, no.20, pp.1926 - 1932
- Indexed
- SCIE
SCOPUS
- Journal Title
- New England Journal of Medicine
- Volume
- 382
- Number
- 20
- Start Page
- 1926
- End Page
- 1932
- URI
- https://scholarworks.bwise.kr/erica/handle/2021.sw.erica/1100
- DOI
- 10.1056/NEJMoa1915872
- ISSN
- 0028-4793
- 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. Copyright © 2020 Massachusetts Medical Society.
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