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Characterization and criteria of embryonic stem and induced pluripotent stem cells for a dopamine replacement therapy

Cooper, Oliver; Parmar, Malin LU and Isacson, Ole (2012) In Progress in Brain Research 200. p.76-265
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... (More)

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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published
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keywords
Animals, Cell Differentiation, Dopaminergic Neurons, Embryonic Stem Cells, Humans, Induced Pluripotent Stem Cells, Mesencephalon, Parkinson Disease, Stem Cell Transplantation, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review
in
Progress in Brain Research
volume
200
pages
12 pages
publisher
Elsevier
external identifiers
  • scopus:84870275738
ISSN
1875-7855
DOI
10.1016/B978-0-444-59575-1.00012-0
language
English
LU publication?
yes
id
2d86bc77-a588-4f7c-b973-7624d09edf11
date added to LUP
2016-11-23 14:28:20
date last changed
2017-04-09 04:51:57
@article{2d86bc77-a588-4f7c-b973-7624d09edf11,
  abstract     = {<p>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.</p>},
  author       = {Cooper, Oliver and Parmar, Malin and Isacson, Ole},
  issn         = {1875-7855},
  keyword      = {Animals,Cell Differentiation,Dopaminergic Neurons,Embryonic Stem Cells,Humans,Induced Pluripotent Stem Cells,Mesencephalon,Parkinson Disease,Stem Cell Transplantation,Journal Article,Research Support, N.I.H., Extramural,Research Support, Non-U.S. Gov't,Review},
  language     = {eng},
  pages        = {76--265},
  publisher    = {Elsevier},
  series       = {Progress in Brain Research},
  title        = {Characterization and criteria of embryonic stem and induced pluripotent stem cells for a dopamine replacement therapy},
  url          = {http://dx.doi.org/10.1016/B978-0-444-59575-1.00012-0},
  volume       = {200},
  year         = {2012},
}