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Related Concept Videos

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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 ALS and FTD with iPSC-derived neurons.

Sebum Lee1, Eric J Huang2

  • 1Department of Pathology, University of California San Francisco, 505 Parnassus Avenue, San Francisco, CA 94143-0502, United States.

Brain Research
|October 15, 2015
PubMed
Summary

Patient-specific stem cells offer new ways to study amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia (FTD), showing promise for drug discovery despite current limitations.

Keywords:
Amyotrophic lateral sclerosis (ALS)Frontotemporal dementia (FTD)Frontotemporal lobar degeneration (FTLD)Induced pluripotent stem cells (iPSCs)

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Area of Science:

  • Neuroscience
  • Genetics
  • Stem Cell Biology

Background:

  • Amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia (FTD) are increasingly viewed as part of a disease spectrum.
  • Existing animal models for ALS and FTD have limitations in reflecting human neurodegenerative processes.

Purpose of the Study:

  • To review the use of induced pluripotent stem cells (iPSCs) in modeling ALS and FTD.
  • To highlight the potential of iPSC-derived neurons for understanding disease mechanisms and drug discovery.

Main Methods:

  • Summarizing studies utilizing patient-specific iPSC-derived neurons.
  • Analyzing the capabilities and limitations of iPSC-based models for ALS and FTD.

Main Results:

  • iPSC-derived neurons can recapitulate key features of ALS and FTD.
  • These models show significant potential for advancing drug discovery efforts.

Conclusions:

  • iPSC-derived neurons represent a powerful tool for modeling ALS and FTD.
  • Further improvements are needed to overcome existing caveats and enhance their efficacy as disease models.