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- Cooper, Oliver, et al.
(författare)
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Characterization and criteria of embryonic stem and induced pluripotent stem cells for a dopamine replacement therapy
- 2012
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Ingår i: Progress in Brain Research. - 1875-7855. ; 200, s. 76-265
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Tidskriftsartikel (refereegranskat)abstract
- Human pluripotent stem cells provide new choices for sources of A9-type dopaminergic (DA) neurons in clinical trials of neural transplantation for patients with Parkinson's disease (PD). For example, "self" and HLA-matched A9 DA neurons may improve the patient-to-patient variability observed in previous clinical trials using fetal DA neurons and obviate the need for long-term immunosuppression in the patient. Normal chromosomal structure and minimal somatic mutations in pluripotent stem cells are necessary criteria for assuring the safe and reproducible transplantation of differentiated DA neurons into patients with PD in clinical trials. However, with these new choices of cell source, the application of pluripotency assays as criteria to ensure pluripotent stem cell quality becomes less relevant. New more relevant standards of quality control, assurance, and function are required. We suggest that quality assurance measures for pluripotent stem cells need to focus upon readouts for authentic midbrain DA neurons, their integration and growth using in vivo assays, and their long-term functional stability.
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| 5. |
- Lindvall, Olle, et al.
(författare)
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Clinical translation of stem cells in neurodegenerative disorders.
- 2012
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Ingår i: Cell Stem Cell. - : Elsevier BV. - 1934-5909. ; 10:2, s. 151-155
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Tidskriftsartikel (refereegranskat)abstract
- Stem cells and their derivatives show tremendous potential for treating many disorders, including neurodegenerative diseases. We discuss here the challenges and potential for the translation of stem-cell-based approaches into treatments for Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.
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| 6. |
- Wray, Selina, et al.
(författare)
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Creation of an Open-Access, Mutation-Defined Fibroblast Resource for Neurological Disease Research
- 2012
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Ingår i: PLoS ONE. - : Public Library of Science (PLoS). - 1932-6203. ; 7:8
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Tidskriftsartikel (refereegranskat)abstract
- Our understanding of the molecular mechanisms of many neurological disorders has been greatly enhanced by the discovery of mutations in genes linked to familial forms of these diseases. These have facilitated the generation of cell and animal models that can be used to understand the underlying molecular pathology. Recently, there has been a surge of interest in the use of patient-derived cells, due to the development of induced pluripotent stem cells and their subsequent differentiation into neurons and glia. Access to patient cell lines carrying the relevant mutations is a limiting factor for many centres wishing to pursue this research. We have therefore generated an open-access collection of fibroblast lines from patients carrying mutations linked to neurological disease. These cell lines have been deposited in the National Institute for Neurological Disorders and Stroke (NINDS) Repository at the Coriell Institute for Medical Research and can be requested by any research group for use in in vitro disease modelling. There are currently 71 mutation-defined cell lines available for request from a wide range of neurological disorders and this collection will be continually expanded. This represents a significant resource that will advance the use of patient cells as disease models by the scientific community.
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