Personalized IPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease

Overview

Journal N Engl J Med

Specialty General Medicine

Date 2020 May 14

PMID 32402162

Citations 223

Authors

Jeffrey S Schweitzer

Bin Song

Todd M Herrington

Tae-Yoon Park

Nayeon Lee

Sanghyeok Ko

Jeha Jeon

Young Cha

Kyungsang Kim

Quanzheng Li

Claire Henchcliffe

Michael Kaplitt

Carolyn Neff

Otto Rapalino

Hyemyung Seo

In-Hee Lee

Jisun Kim

Taewoo Kim

Gregory A Petsko

Jerome Ritz

Bruce M Cohen

Sek-Won Kong

Pierre Leblanc

Bob S Carter

Kwang-Soo Kim

Affiliations

Soon will be listed here.

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.).

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