Using Human-Induced Pluripotent Stem Cells to Model Monogenic Metabolic Disorders of the Liver

Maria Paulina Ordonez; Lawrence S.B. Goldstein

doi:10.1055/s-0032-1329898

Seminars in Liver Disease, Inhaltsverzeichnis

Semin Liver Dis 2012; 32(04): 298-306
DOI: 10.1055/s-0032-1329898

Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Using Human-Induced Pluripotent Stem Cells to Model Monogenic Metabolic Disorders of the Liver

Maria Paulina Ordonez

¹Department of Pediatric Gastroenterology, Hepatology and Nutrition, University of California, La Jolla, San Diego, California

,

Lawrence S.B. Goldstein

²Department of Cellular and Molecular Medicine, University of California, La Jolla, San Diego, California

› Institutsangaben

Abstract

A crucial problem in liver disease biology and a major obstacle to the development of new therapies is the inability to conduct mechanistic studies of live human hepatocytes. Liver tissue from patients is difficult to obtain and only reveals the disease aftermath, while animal models lack the significant genetic diversity of humans. Monogenic metabolic disorders of the liver are an ideal platform to explore the complex gene–environment interactions and the role of genetic variation in the onset and progression of liver disease. Human induced pluripotent stem cell (hIPSC) technology provides an unprecedented opportunity to generate live cellular models of disease for therapeutic candidate discovery and cell replacement therapy. In this review, we discuss the potential of hIPSC to increase our understanding of human disease with a focus on the current efforts to model metabolic diseases of the liver and to generate suitable populations of human hepatocytes for cell transplantation.

Keywords

human-induced pluripotent stem cells - hepatocyte differentiation - disease modeling - inherited liver metabolic disorders - cell transplantation

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