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Related Concept Videos

Induced Pluripotent Stem Cells01:13

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Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore...
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Induced Pluripotent Stem Cells01:06

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Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic...
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Drug Discovery: Overview01:26

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Embryonic stem (ES) cells are undifferentiated pluripotent cells, meaning they can produce any cell type in the body. This gives them tremendous potential in science and medicine since they can generate specific cell types for use in research or to replace body cells lost due to damage or disease.
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Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
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Stem cells are undifferentiated cells that divide and produce more stem cells or progenitor cells that differentiate into mature, specialized cell types. All the cells in the body are generated from stem cells in the early embryo, but small populations of stem cells are also present in many adult tissues including the bone marrow, brain, skin, and gut. These adult stem cells typically produce the various cell types found in that tissue—to replace cells that are damaged or to continuously...
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Updated: Feb 7, 2026

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery
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Pluripotent Stem Cell Platforms for Drug Discovery.

Kevin G Chen1, Barbara S Mallon1, Kyeyoon Park1

  • 1NIH Stem Cell Characterization Facility, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.

Trends in Molecular Medicine
|July 15, 2018
PubMed
Summary
This summary is machine-generated.

Human pluripotent stem cells (hPSCs) advance drug discovery, but current methods face limitations. This review analyzes 2D, 3D, and organoid systems to improve human pluripotent stem cell-based drug discovery (hPDD) for conditions like cystic fibrosis.

Keywords:
ATP-binding cassetteCFTRPluripotent stem cellscystic fibrosisdifferentiationdrug discoveryorganoids

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Area of Science:

  • Biomedical Sciences
  • Stem Cell Biology
  • Pharmacology

Background:

  • Human pluripotent stem cells (hPSCs) and their derivatives are pivotal in proof-of-principle drug discoveries.
  • Current human pluripotent stem cell-based drug discovery (hPDD) strategies are hindered by conceptual biases and technological constraints.
  • Limitations include cell-culture dimensionality, maturity, functionality, experimental variability, and data reproducibility.

Purpose of the Study:

  • To critically evaluate existing hPSC-based drug discovery (hPDD) systems.
  • To analyze 2D-monolayers, 3D cultures, and organoids within hPDD.
  • To discuss drug discovery, repurposing mechanisms, and the role of membrane drug transporters in hPDD, using cystic fibrosis as a model.

Main Methods:

  • Systematic review and analysis of representative hPSC-based drug discovery (hPDD) systems.
  • Dissection of 2D-monolayer, 3D culture, and organoid methodologies.
  • Examination of drug discovery and repurposing mechanisms, focusing on membrane drug transporters.

Main Results:

  • Analysis reveals inherent limitations in current hPDD approaches regarding dimensionality, cell maturity, and reproducibility.
  • Different hPSC culture methods (2D, 3D, organoids) present unique advantages and disadvantages for drug discovery.
  • Membrane drug transporters play a crucial role in tissue maturation and hPDD efficacy.

Conclusions:

  • Optimizing hPSC-based drug discovery (hPDD) requires addressing current technological and conceptual limitations.
  • Advancements in 3D culture and organoid models show promise for more physiologically relevant drug screening.
  • Understanding membrane drug transporter functions is essential for refining hPDD strategies, particularly for genetic diseases like cystic fibrosis.