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Related Concept Videos

Stem Cell Culture01:17

Stem Cell Culture

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Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
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Embryonic Stem Cells00:57

Embryonic Stem Cells

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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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EPS and iPS Cells in Disease Research01:21

EPS and iPS Cells in Disease Research

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Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
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Lineage Commitment01:21

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Commitment is the  process whereby stem cells:
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Related Experiment Video

Updated: Jul 19, 2025

A Combinatorial Single-cell Approach to Characterize the Molecular and Immunophenotypic Heterogeneity of Human Stem and Progenitor Populations
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Functional genomics in stem cell models: considerations and applications.

Kaivalya Shevade1,2, Sailaja Peddada1,2, Karl Mader1,2

  • 1Laboratory for Genomics Research, San Francisco, CA, United States.

Frontiers in Cell and Developmental Biology
|August 9, 2023
PubMed
Summary
This summary is machine-generated.

Human pluripotent stem cell differentiation protocols advance disease modeling. CRISPR-Cas9 technology enables precise gene editing in induced pluripotent stem cells (iPSCs) for drug discovery and mechanism elucidation.

Keywords:
CRISPR screeningdrug discoveryfunctional genomicshuman diseaseiPSC (induced pluripotent stem cell)iPSC-derived models

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Area of Science:

  • Stem cell biology
  • Genomics
  • Biotechnology

Background:

  • Human pluripotent stem cell differentiation protocols have significantly advanced over the past two decades, enhancing cell type specificity and tissue complexity.
  • The generation of induced pluripotent stem cells (iPSCs) from patients allows for disease modeling in relevant cellular contexts, aiding the study of disease etiology and progression.
  • iPSC-derived models are increasingly used for drug screening to identify novel therapies and therapeutic targets.

Purpose of the Study:

  • To review current technologies in induced pluripotent stem cell (iPSC) disease modeling.
  • To explore CRISPR-based functional genomics screens for identifying genetic modifiers and therapeutic targets.
  • To discuss practical considerations for implementing these technologies across various applications and disease areas.

Main Methods:

  • Utilizing CRISPR-Cas9 technology, including CRISPR interference (CRISPRi) and CRISPR activation (CRISPRa), for precise gene editing in iPSCs.
  • Employing CRISPR knockout, CRISPRi, and CRISPRa screens for systematic and unbiased identification of genetic modifiers.
  • Reviewing existing literature on iPSC disease models and CRISPR screens.

Main Results:

  • CRISPR-Cas9 technology, including CRISPRi and CRISPRa, enables advanced gene editing beyond single gene modifications.
  • CRISPR screens in iPSC models have identified genetic modifiers that synergize or antagonize disease mutations.
  • These screens have led to the identification of disease mechanisms and potential therapeutic targets.

Conclusions:

  • Advancements in iPSC differentiation and CRISPR-based functional genomics accelerate disease mechanism discovery and drug development.
  • Despite technical challenges, CRISPR screens in iPSC models offer powerful insights for therapeutic strategies.
  • The integration of these technologies holds significant promise for personalized medicine and the discovery of novel therapies.