Literature DB >> 32402162

Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease.

Jeffrey S Schweitzer1, Bin Song1, Todd M Herrington1, Tae-Yoon Park1, Nayeon Lee1, Sanghyeok Ko1, Jeha Jeon1, Young Cha1, Kyungsang Kim1, Quanzheng Li1, Claire Henchcliffe1, Michael Kaplitt1, Carolyn Neff1, Otto Rapalino1, Hyemyung Seo1, In-Hee Lee1, Jisun Kim1, Taewoo Kim1, Gregory A Petsko1, Jerome Ritz1, Bruce M Cohen1, Sek-Won Kong1, Pierre Leblanc1, Bob S Carter1, Kwang-Soo Kim1.   

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.).
Copyright © 2020 Massachusetts Medical Society.

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Year:  2020        PMID: 32402162      PMCID: PMC7288982          DOI: 10.1056/NEJMoa1915872

Source DB:  PubMed          Journal:  N Engl J Med        ISSN: 0028-4793            Impact factor:   91.245


  24 in total

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