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Related Concept Videos

iPS Cell Differentiation01:22

iPS Cell Differentiation

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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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EPS and iPS Cells in Disease Research01:21

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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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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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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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Embryonic Stem Cells00:58

Embryonic Stem Cells

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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 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.
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
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Related Experiment Video

Updated: Mar 2, 2026

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
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Stem Cell Technology for (Epi)genetic Brain Disorders.

Renzo J M Riemens1,2,3, Edilene S Soares1, Manel Esteller1,4,5

  • 1Cancer Epigenetics and Biology Program (PEBC), Bellvitge Biomedical Research Institute (IDIBELL), Duran i Reynals Hospital, 3rd floor, Gran Via de L'Hospitalet 199-203, L'Hospitalet de Llobregat, Barcelona, Catalonia, 08908, Spain.

Advances in Experimental Medicine and Biology
|May 20, 2017
PubMed
Summary

Human stem cell technology offers a powerful new approach for studying and treating (epi)genetic brain disorders. These models provide better humanized systems for disease research and therapeutic development.

Keywords:
Brain disordersDisease modelingDrug screeningEpigeneticsRegenerative medicineStem cellsiPSCs

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Area of Science:

  • Neuroscience
  • Stem Cell Biology
  • Genetics

Background:

  • Effective treatments for many (epi)genetic brain disorders are lacking due to limitations in current disease models.
  • Existing animal and cellular models inadequately represent human brain pathologies, hindering translational research.
  • A deeper understanding of multifactorial causes and (epi)genetic alterations is crucial for mechanistic insights.

Purpose of the Study:

  • To demonstrate the validity of human stem cell-based models for studying brain disorders.
  • To explore stem cell applications in disease modeling and therapeutic interventions.
  • To highlight the utility of stem cells for various human brain disorders with genetic bases.

Main Methods:

  • Utilizing human stem cell technology to generate neural cells and precursors in vitro.
  • Developing humanized model systems for studying (epi)genetic brain disorders.
  • Applying stem cell derivatives for drug screening and regenerative medicine.

Main Results:

  • Human stem cell-derived models provide a viable platform for in vitro disease modeling.
  • Stem cells offer an inexhaustible source of neural cells for research and therapy.
  • These models facilitate high-throughput drug and toxicity testing.

Conclusions:

  • Human stem cell-based models represent a significant advancement in understanding and treating (epi)genetic brain disorders.
  • Stem cell technology holds promise for personalized medicine and regenerative therapies.
  • This approach is applicable to a range of disorders including Parkinson's, Alzheimer's, Fragile X, Angelman, Prader-Willi, and Rett syndromes.