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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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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. |
format | Online Article Text |
id | pubmed-9030659 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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