
Andreas Heuer
Research team manager

Human ESC-Derived Dopamine Neurons Show Similar Preclinical Efficacy and Potency to Fetal Neurons when Grafted in a Rat Model of Parkinson's Disease.
Author
Summary, in English
Considerable progress has been made in generating fully functional and transplantable dopamine neurons from human embryonic stem cells (hESCs). Before these cells can be used for cell replacement therapy in Parkinson's disease (PD), it is important to verify their functional properties and efficacy in animal models. Here we provide a comprehensive preclinical assessment of hESC-derived midbrain dopamine neurons in a rat model of PD. We show long-term survival and functionality using clinically relevant MRI and PET imaging techniques and demonstrate efficacy in restoration of motor function with a potency comparable to that seen with human fetal dopamine neurons. Furthermore, we show that hESC-derived dopamine neurons can project sufficiently long distances for use in humans, fully regenerate midbrain-to-forebrain projections, and innervate correct target structures. This provides strong preclinical support for clinical translation of hESC-derived dopamine neurons using approaches similar to those established with fetal cells for the treatment of Parkinson's disease.
Department/s
- Developmental and Regenerative Neurobiology
- Stem Cell Center
- Neurobiology
- StemTherapy: National Initiative on Stem Cells for Regenerative Therapy
- MultiPark: Multidisciplinary research focused on Parkinson´s disease
Publishing year
2014
Language
English
Pages
653-665
Publication/Series
Cell Stem Cell
Volume
15
Issue
5
Full text
- Available as PDF - 15 MB
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Links
Document type
Journal article
Publisher
Cell Press
Topic
- Cell Biology
Status
Published
Research group
- Developmental and Regenerative Neurobiology
- Neurobiology
ISBN/ISSN/Other
- ISSN: 1934-5909