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EPS and iPS Cells in Disease Research

EPS and iPS Cells in Disease Research

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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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 called induced pluripotent stem...

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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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Induced Pluripotent Stem Cells

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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 called induced pluripotent stem...

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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Phenotypic Profiling of Human Stem Cell-Derived Midbrain Dopaminergic Neurons

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Published on: July 7, 2023

Modeling Parkinson's disease using induced pluripotent stem cells.

Blake Byers¹, Hsiao-lu Lee, Renee Reijo Pera

¹Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Palo Alto, CA, USA.

Current Neurology and Neuroscience Reports

|April 28, 2012

Summary

Patient-derived induced pluripotent stem cells (iPSCs) create Parkinson's disease (PD) models. These models show early disease signs in dopaminergic neurons, aiding in developing new diagnostics and therapeutics.

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Area of Science:

Neuroscience
Stem Cell Biology
Genetics

Background:

Parkinson's disease (PD) research is limited by the lack of accessible human dopaminergic (DA) neurons.
Induced pluripotent stem cells (iPSCs) offer a promising avenue for modeling PD and understanding its molecular mechanisms.

Purpose of the Study:

To generate and characterize patient-derived iPSC lines modeling familial Parkinson's disease.
To investigate the utility of these iPSC-derived DA neurons for studying PD pathogenesis and therapeutic development.

Main Methods:

Generated two patient-derived iPSC lines: one with a LRRK2 p.G2019S mutation and another with SNCA gene triplication.
Differentiated iPSCs into DA neurons and assessed their molecular and functional characteristics.
Exposed iPSC-derived DA neurons to various stressors to evaluate cellular vulnerability.

Main Results:

Both LRRK2-G2019S and SNCA-triplication iPSC-derived DA neurons exhibited increased oxidative stress gene expression and α-synuclein protein levels.
LRRK2-G2019S DA neurons showed increased sensitivity to caspase-3 activation. SNCA-triplication DA neurons formed ubiquitin-positive puncta and were more susceptible to oxidative stress.
These findings indicate early PD-related phenotypes in the developed iPSC models.

Conclusions:

Patient-derived iPSC lines carrying LRRK2 or SNCA mutations provide valuable models for Parkinson's disease.
These models exhibit key cellular phenotypes relevant to PD pathogenesis.
They hold potential for advancing disease diagnostics and the development of novel therapeutic strategies.