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Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells

Serving as the metabolic hub of the human body, the liver is a vital organ that performs a variety of important physiological functions. Although known for its regenerative potential, it remains vulnerable to a variety of diseases. Despite decades of research, liver disease remains a leading cause o...

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Detalles Bibliográficos
Autores principales: Blaszkiewicz, Josef, Duncan, Stephen A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9030659/
https://www.ncbi.nlm.nih.gov/pubmed/35456379
http://dx.doi.org/10.3390/genes13040573
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author Blaszkiewicz, Josef
Duncan, Stephen A.
author_facet Blaszkiewicz, Josef
Duncan, Stephen A.
author_sort Blaszkiewicz, Josef
collection PubMed
description Serving as the metabolic hub of the human body, the liver is a vital organ that performs a variety of important physiological functions. Although known for its regenerative potential, it remains vulnerable to a variety of diseases. Despite decades of research, liver disease remains a leading cause of mortality in the United States with a multibillion-dollar-per-year economic burden. Prior research with model systems, such as primary hepatocytes and murine models, has provided many important discoveries. However, progress has been impaired by numerous obstacles associated with these models. In recent years, induced pluripotent stem cell (iPSC)-based systems have emerged as advantageous platforms for studying liver disease. Benefits, including preserved differentiation and physiological function, amenability to genetic manipulation via tools such as CRISPR/Cas9, and availability for high-throughput screening, make these systems increasingly attractive for both mechanistic studies of disease and the identification of novel therapeutics. Although limitations exist, recent studies have made progress in ameliorating these issues. In this review, we discuss recent advancements in iPSC-based models of liver disease, including improvements in model system construction as well as the use of high-throughput screens for genetic studies and drug discovery.
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spelling pubmed-90306592022-04-23 Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells Blaszkiewicz, Josef Duncan, Stephen A. Genes (Basel) Review Serving as the metabolic hub of the human body, the liver is a vital organ that performs a variety of important physiological functions. Although known for its regenerative potential, it remains vulnerable to a variety of diseases. Despite decades of research, liver disease remains a leading cause of mortality in the United States with a multibillion-dollar-per-year economic burden. Prior research with model systems, such as primary hepatocytes and murine models, has provided many important discoveries. However, progress has been impaired by numerous obstacles associated with these models. In recent years, induced pluripotent stem cell (iPSC)-based systems have emerged as advantageous platforms for studying liver disease. Benefits, including preserved differentiation and physiological function, amenability to genetic manipulation via tools such as CRISPR/Cas9, and availability for high-throughput screening, make these systems increasingly attractive for both mechanistic studies of disease and the identification of novel therapeutics. Although limitations exist, recent studies have made progress in ameliorating these issues. In this review, we discuss recent advancements in iPSC-based models of liver disease, including improvements in model system construction as well as the use of high-throughput screens for genetic studies and drug discovery. MDPI 2022-03-24 /pmc/articles/PMC9030659/ /pubmed/35456379 http://dx.doi.org/10.3390/genes13040573 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Blaszkiewicz, Josef
Duncan, Stephen A.
Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title_full Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title_fullStr Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title_full_unstemmed Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title_short Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells
title_sort advancements in disease modeling and drug discovery using ipsc-derived hepatocyte-like cells
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9030659/
https://www.ncbi.nlm.nih.gov/pubmed/35456379
http://dx.doi.org/10.3390/genes13040573
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