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Related Concept Videos

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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Modeling, a key technique in therapy, uses observational learning to help clients acquire and practice new skills by watching therapists demonstrate desired behaviors. This approach, rooted in Albert Bandura's concept of vicarious learning, plays a significant role in therapeutic interventions for various psychological conditions, including social anxiety, ADHD, and depression.
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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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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).
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Autism spectrum disorder (ASD) is a neurodevelopmental condition marked by persistent deficits in social communication and interaction alongside restrictive and repetitive behaviors or interests. ASD is sometimes accompanied by intellectual impairment.
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Autism spectrum disorders and disease modeling using stem cells.

Anita Brito1,2, Fabiele Baldino Russo1,2, Alysson Renato Muotri3

  • 1Department of Surgery, Stem Cell Laboratory, University of São Paulo, São Paulo, 05508-270, Brazil.

Cell and Tissue Research
|September 18, 2017
PubMed
Summary

Induced pluripotent stem cell technology offers a novel approach to understanding autism spectrum disorders (ASD). Modeling central nervous system cells in vitro aids in comprehending ASD pathology and personalizing treatments.

Keywords:
ASDAutism spectrum disordersDisease modelingStem cellsiPSC

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Area of Science:

  • Neuroscience
  • Developmental Biology
  • Genetics

Background:

  • Autism spectrum disorders (ASD) are complex neurodevelopmental disabilities impacting communication, socialization, and behavior.
  • ASD presents a wide spectrum of severity and associated comorbidities, including intellectual disability and unique talents.
  • Significant variability exists in clinical presentation, from severe communication impairments to advanced early language skills.

Purpose of the Study:

  • To explore the application of induced pluripotent stem cell (iPSC) technology in modeling autism spectrum disorders.
  • To investigate how in vitro modeling of central nervous system cells can elucidate ASD pathology.
  • To determine the potential of iPSC-based models for understanding ASD variability and personalizing treatments.

Main Methods:

  • Utilizing induced pluripotent stem cell (iPSC) technology to generate neural cells in vitro.
  • Modeling both syndromic and non-syndromic forms of autism in a laboratory setting.
  • Analyzing cellular and molecular characteristics of iPSC-derived neurons to understand disease mechanisms.

Main Results:

  • iPSC technology enables the creation of relevant cellular models for studying autism spectrum disorders.
  • These models can recapitulate aspects of ASD pathology and highlight underlying biological differences.
  • The variability observed in ASD patients can be potentially mirrored in iPSC-derived neural cultures.

Conclusions:

  • Induced pluripotent stem cell technology is a valuable tool for advancing the understanding of autism spectrum disorders.
  • In vitro modeling facilitates the study of ASD's complex pathology and spectrum of symptoms.
  • This approach holds promise for the development of personalized therapeutic strategies for individuals with ASD.