Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease.
N Engl J Med
; 382(20): 1926-1932, 2020 05 14.
Article
em En
| MEDLINE
| ID: mdl-32402162
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.).
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doença de Parkinson
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Células-Tronco Pluripotentes Induzidas
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Neurônios Dopaminérgicos
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Parte Compacta da Substância Negra
Tipo de estudo:
Observational_studies
/
Prognostic_studies
Limite:
Aged
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Animals
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Humans
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Male
Idioma:
En
Revista:
N Engl J Med
Ano de publicação:
2020
Tipo de documento:
Article
País de publicação:
Estados Unidos