The DOI matches its Crossref record; 31 of the 31 references it cites were found in a registry or an index.
Human ESC-Derived Dopamine Neurons Show Similar Preclinical Efficacy and Potency to Fetal Neurons when Grafted in a Rat Model of Parkinson’s Disease
Open accessAbstract
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.
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Glia-to-Neuron Conversion by CRISPR-CasRx Alleviates Symptoms of Neurological Disease in Mice
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Human Trials of Stem Cell-Derived Dopamine Neurons for Parkinson’s Disease: Dawn of a New Era
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Metadata
- Authors
- Grealish, S., Diguet, E., Kirkeby, A., Mattsson, B., Heuer, A., Bramoulle, Y., Van Camp, N., Perrier, A. L., Hantraye, P., Björklund, A., & Parmar, M.
- Venue
- Cell Stem Cell
- Publisher
- Elsevier BV
- Published
- 2014-11
- Volume
- 15
- Issue
- 5
- Pages
- 653-665
- ISSN
- 1875-9777 (ISSN-L)
- 1934-5909
- OpenAlex
- W2057335179
- Cited by
- 447
- References
- 31
- FWCI
- 15.82
- Type
- journal article
- Language
- en
- Open access
- hybrid
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