Modeling Inborn Errors of Hepatic Metabolism Using Induced Pluripotent Stem Cells.

ABSTRACT: Inborn errors of hepatic metabolism are because of deficiencies commonly within a single enzyme as a consequence of heritable mutations in the genome. Individually such diseases are rare, but collectively they are common. Advances in genome-wide association studies and DNA sequencing have helped researchers identify the underlying genetic basis of such diseases. Unfortunately, cellular and animal models that accurately recapitulate these inborn errors of hepatic metabolism in the laboratory have been lacking. Recently, investigators have exploited molecular techniques to generate induced pluripotent stem cells from patients' somatic cells. Induced pluripotent stem cells can differentiate into a wide variety of cell types, including hepatocytes, thereby offering an innovative approach to un

SUBMITTER: Pournasr B 

PROVIDER: S-EPMC5824724 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature