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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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Related Experiment Video

Updated: Oct 7, 2025

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Stem Cell-Based Disease Models for Inborn Errors of Immunity.

Aline Zbinden1, Kirsten Canté-Barrett1, Karin Pike-Overzet1

  • 1Department of Immunology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.

Cells
|January 11, 2022
PubMed
Summary

Human hematopoietic stem cells (hHSCs) offer gene therapy potential for immune diseases. Current stem cell models, while valuable, face challenges in accurately reflecting disease complexity for developing next-generation therapies.

Keywords:
hematopoietic stem cellsimmune deficiencyinborn errors of immunityinduced pluripotent stem cellsprimary immune deficiency

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Area of Science:

  • * Stem Cell Biology and Regenerative Medicine
  • * Immunology and Gene Therapy
  • * Human Pathophysiology

Background:

  • * Human hematopoietic stem cells (hHSCs) possess self-renewal and multi-lineage differentiation capabilities, crucial for gene therapy.
  • * Inborn errors of immunity (IEI) are primary targets for hHSC-based gene therapies.
  • * Accurate disease models are essential for advancing hHSC-based therapeutic development.

Purpose of the Study:

  • * To discuss challenges in hHSC disease modeling, including developmental origin and heterogeneity.
  • * To review existing in vitro and in vivo models for inborn errors of immunity.
  • * To critically evaluate the strengths and limitations of current stem cell-based models for IEI.

Main Methods:

  • * Review of literature on induced pluripotent stem cell (iPSC) technology.
  • * Analysis of models utilizing adult hHSCs.
  • * Critical assessment of in vitro and in vivo experimental models for IEI.

Main Results:

  • * Significant challenges exist in hHSC developmental origin and heterogeneity, impacting disease modeling.
  • * Both iPSC-derived and adult hHSC-based models present distinct advantages and limitations.
  • * Current models provide valuable insights but require further refinement for comprehensive IEI study.

Conclusions:

  • * Stem cell-based models are indispensable for advancing gene therapy for IEI.
  • * Overcoming challenges in developmental origin and heterogeneity is key for improved models.
  • * Future stem cell models will be critical for developing next-generation IEI therapies.