Document Detail


Modelling human disease with pluripotent stem cells.
MedLine Citation:
PMID:  23444871     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Recent progress in the field of cellular reprogramming has opened up the doors to a new era of disease modelling, as pluripotent stem cells representing a myriad of genetic diseases can now be produced from patient tissue. These cells can be expanded and differentiated to produce a potentially limitless supply of the affected cell type, which can then be used as a tool to improve understanding of disease mechanisms and test therapeutic interventions. This process requires high levels of scrutiny and validation at every stage, but international standards for the characterisation of pluripotent cells and their progeny have yet to be established. Here we discuss the current state of the art with regard to modelling diseases affecting the ectodermal, mesodermal and endodermal lineages, focussing on studies which have demonstrated a disease phenotype in the tissue of interest. We also discuss the utility of pluripotent cell technology for the modelling of cancer and infectious disease. Finally, we spell out the technical and scientific challenges which must be addressed if the field is to deliver on its potential and produce improved patient outcomes in the clinic.
Authors:
Richard Siller; Sebastian Greenhough; In-Hyun Park; Gareth J Sullivan
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Current gene therapy     Volume:  13     ISSN:  1875-5631     ISO Abbreviation:  Curr Gene Ther     Publication Date:  2013 Apr 
Date Detail:
Created Date:  2013-03-27     Completed Date:  2013-09-23     Revised Date:  2013-10-17    
Medline Journal Info:
Nlm Unique ID:  101125446     Medline TA:  Curr Gene Ther     Country:  Netherlands    
Other Details:
Languages:  eng     Pagination:  99-110     Citation Subset:  IM    
Affiliation:
Stem Cell Epigenetics Laboratory, Institute of Basic Medical Sciences, Faculty of Medicine, University of Oslo, P.O. Box 1112, Blindern, 0317 Oslo, Norway.
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MeSH Terms
Descriptor/Qualifier:
Cell Differentiation*
Cell Lineage
Embryonic Stem Cells / cytology,  transplantation
Humans
Neurodegenerative Diseases / therapy*
Nuclear Reprogramming / genetics*
Pluripotent Stem Cells / cytology*,  transplantation
Translational Medical Research
Grant Support
ID/Acronym/Agency:
GM0099130-01/GM/NIGMS NIH HHS; P01 GM099130/GM/NIGMS NIH HHS; UL1 RR025750/RR/NCRR NIH HHS
Comments/Corrections

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