상위피인용논문
Sloan Kettering Cancer Center, Weill Cornell Medicine
Tae Wan Kim 1 2 †, So Yeon Koo 1 2 3 †, Lorenz Studer 1 2 *
1The Center for Stem Cell Biology, Developmental Biology Program, Sloan-Kettering Institute for Cancer Research, New York, NY, United States.
2Developmental Biology Program, Sloan-Kettering Institute for Cancer Research, New York, NY, United States.
3Neuroscience Graduate Program of Weill Cornell Graduate School of Biomedical Sciences, Weill Cornell Medicine, New York, NY, United States.
†These authors have contributed equally to this work
*Corresponding author: correspondence to Lorenz Studer
Abstract
In Parkinson’s disease (PD), there are currently no effective therapies to prevent or slow down disease progression. Cell replacement therapy using human pluripotent stem cell (hPSC)-derived dopamine neurons holds considerable promise. It presents a novel, regenerative strategy, building on the extensive history of fetal tissue grafts and capturing the potential of hPSCs to serve as a scalable and standardized cell source. Progress in establishing protocols for the direct differentiation to midbrain dopamine (mDA) neurons from hPSC have catalyzed the development of cell-based therapies for PD. Consequently, several groups have derived clinical-grade mDA neuron precursors under clinical good manufacture practice condition, which are progressing toward clinical testing in PD patients. Here we will review the current status of the field, discuss the remaining key challenges, and highlight future areas for further improvements of hPSC-based technologies in the clinical translation to PD.
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