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Clinical translation of stem cell transplantation in Parkinson's disease.

Lindvall, Olle LU (2016) In Journal of Internal Medicine 279(1). p.30-40
Abstract
In Parkinson's disease (PD), the main pathology underlying the motor symptoms is a loss of nigrostriatal dopaminergic neurons. Clinical trials of intrastriatal transplantation of human foetal mesencephalic tissue have shown that the grafted dopaminergic neurons re-innervate the striatum, restore striatal dopamine release and, in some cases, induce major, long-lasting improvement of motor function. However, nonmotor symptoms originating from degeneration outside the striatum or in nondopaminergic systems are not alleviated by intrastriatal implantation of dopaminergic neurons. Stem cells and reprogrammed cells could potentially be used to produce dopaminergic neurons for transplantation in patients with PD. Recent studies demonstrate that... (More)
In Parkinson's disease (PD), the main pathology underlying the motor symptoms is a loss of nigrostriatal dopaminergic neurons. Clinical trials of intrastriatal transplantation of human foetal mesencephalic tissue have shown that the grafted dopaminergic neurons re-innervate the striatum, restore striatal dopamine release and, in some cases, induce major, long-lasting improvement of motor function. However, nonmotor symptoms originating from degeneration outside the striatum or in nondopaminergic systems are not alleviated by intrastriatal implantation of dopaminergic neurons. Stem cells and reprogrammed cells could potentially be used to produce dopaminergic neurons for transplantation in patients with PD. Recent studies demonstrate that standardized preparations of dopaminergic neurons of the correct substantia nigra phenotype can be generated from human embryonic stem cells in large numbers, and they will soon be available for patient application. In addition, dopaminergic neurons derived from human induced pluripotent stem cells are being considered for clinical translation. Important challenges include the demonstration of potency (growth capacity and functional efficacy) and safety of the generated dopaminergic neurons in preclinical animal models. The dopaminergic neurons should subsequently be tested, using optimal patient selection and cell preparation and transplantation procedures, in controlled clinical studies. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Journal of Internal Medicine
volume
279
issue
1
pages
30 - 40
publisher
Wiley-Blackwell Publishing Ltd
external identifiers
  • pmid:26332959
  • wos:000366606200003
  • scopus:84949625733
ISSN
1365-2796
DOI
10.1111/joim.12415
language
English
LU publication?
yes
id
ddb3b2f0-4c32-42c0-b2a8-154412e881bb (old id 8043412)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/26332959?dopt=Abstract
date added to LUP
2015-10-04 19:17:44
date last changed
2017-09-24 03:18:25
@article{ddb3b2f0-4c32-42c0-b2a8-154412e881bb,
  abstract     = {In Parkinson's disease (PD), the main pathology underlying the motor symptoms is a loss of nigrostriatal dopaminergic neurons. Clinical trials of intrastriatal transplantation of human foetal mesencephalic tissue have shown that the grafted dopaminergic neurons re-innervate the striatum, restore striatal dopamine release and, in some cases, induce major, long-lasting improvement of motor function. However, nonmotor symptoms originating from degeneration outside the striatum or in nondopaminergic systems are not alleviated by intrastriatal implantation of dopaminergic neurons. Stem cells and reprogrammed cells could potentially be used to produce dopaminergic neurons for transplantation in patients with PD. Recent studies demonstrate that standardized preparations of dopaminergic neurons of the correct substantia nigra phenotype can be generated from human embryonic stem cells in large numbers, and they will soon be available for patient application. In addition, dopaminergic neurons derived from human induced pluripotent stem cells are being considered for clinical translation. Important challenges include the demonstration of potency (growth capacity and functional efficacy) and safety of the generated dopaminergic neurons in preclinical animal models. The dopaminergic neurons should subsequently be tested, using optimal patient selection and cell preparation and transplantation procedures, in controlled clinical studies.},
  author       = {Lindvall, Olle},
  issn         = {1365-2796},
  language     = {eng},
  number       = {1},
  pages        = {30--40},
  publisher    = {Wiley-Blackwell Publishing Ltd},
  series       = {Journal of Internal Medicine},
  title        = {Clinical translation of stem cell transplantation in Parkinson's disease.},
  url          = {http://dx.doi.org/10.1111/joim.12415},
  volume       = {279},
  year         = {2016},
}