Abstract
Stem cell treatments for neurodegenerative diseases are expected to reach clin. trials soon. Most of the approaches currently under development involve transplantation of immature progenitors that subsequently undergo phenotypic and functional maturation in vivo, and predicting the long-term graft outcome already at the progenitor stage remains a challenge. Here, we took an unbiased approach to identify predictive markers expressed in dopamine neuron progenitors that correlate with graft outcome in an animal model of Parkinson's disease through gene expression anal. of >30 batches of grafted human embryonic stem cell (hESC)-derived progenitors. We found that many of the commonly used markers did not accurately predict in vivo subtype-specific maturation. Instead, we identified a specific set of markers assocd. with the caudal midbrain that correlate with high dopaminergic yield after transplantation in vivo. Using these markers, we developed a good manufg. practice (GMP) differentiation protocol for highly efficient and reproducible prodn. of transplantable dopamine progenitors from hESCs. [on SciFinder(R)]
Original language | English |
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Journal | Cell Stem Cell |
Volume | 20 |
Issue number | 1 |
Pages (from-to) | 135-148 |
Number of pages | 14 |
ISSN | 1875-9777 |
DOIs | |
Publication status | Published - 2017 |
Externally published | Yes |
Bibliographical note
M1 - Copyright (C) 2018 American Chemical Society (ACS). All Rights Reserved.CAPLUS AN 2016:1779232(Journal; Online Computer File)
Keywords
- biomarker cell differentiation hESC transplant Parkinson Disease