Concise Review: Cell Therapies for Hereditary Metabolic Liver Diseases—Concepts, Clinical Results, and Future Developments

The concept of cell‐based therapies for inherited metabolic liver diseases has been introduced for now more than 40 years in animal experiments, but controlled clinical data in humans are still not available. In the era of dynamic developments in stem cell science, the “right” cell for transplantati...

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Published inStem cells (Dayton, Ohio) Vol. 33; no. 4; pp. 1055 - 1062
Main Authors Cantz, Tobias, Sharma, Amar Deep, Ott, Michael
Format Journal Article
LanguageEnglish
Published United States Oxford University Press 01.04.2015
Subjects
Animals
Cell Differentiation - physiology
Cell- and Tissue-Based Therapy - methods
Cell- and Tissue-Based Therapy - trends
Cellular therapy
Clinical translation
Forecasting
Hepatic stem cells
Hepatocyte differentiation
Hepatocytes - transplantation
Humans
Liver
Liver diseases
Liver Diseases - metabolism
Liver Diseases - pathology
Liver Diseases - therapy
Liver Regeneration - physiology
Metabolism
Pluripotent stem cells
Progenitor cells
Stem Cell Transplantation - methods
Stem Cell Transplantation - trends
Stem cells
Transplants & implants
Hepatocyte differentiation
Liver
Cellular therapy
Clinical translation
Hepatic stem cells
Pluripotent stem cells
Progenitor cells
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Summary:The concept of cell‐based therapies for inherited metabolic liver diseases has been introduced for now more than 40 years in animal experiments, but controlled clinical data in humans are still not available. In the era of dynamic developments in stem cell science, the “right” cell for transplantation is considered as an important key for successful treatment. Do we aim to transplant mature hepatocytes or do we consider the liver as a stem/progenitor‐driven organ and replenish the diseased liver with genetically normal stem/progenitor cells? Although conflicting results from cell tracing and transplantation experiments have recently emerged about the existence and role of stem/progenitor cells in the liver, their overall contribution to parenchymal cell homeostasis and tissue repair is limited. Accordingly, engraftment and repopulation efficacies of extrahepatic and liver‐derived stem/progenitor cell types are considered to be lower compared to mature hepatocytes. On the basis of these results, we will discuss the current clinical cell transplantation programs for inherited metabolic liver diseases and future developments in liver cell therapy. Stem Cells 2015;33:1055–1062
Bibliography:available online without subscription through the open access option.
The copyright line for this article was changed on 30 June after original online publication.
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ISSN:1066-5099
1549-4918
DOI:10.1002/stem.1920