Phenotyping Neurodegeneration in Human iPSCs

Induced pluripotent stem cell (iPSC) technology holds promise for modeling neurodegenerative diseases. Traditional approaches for disease modeling using animal and cellular models require knowledge of disease mutations. However, many patients with neurodegenerative diseases do not have a known genet...

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Bibliographic Details
Main Authors: Li, Jonathan, Fraenkel, Ernest
Other Authors: Massachusetts Institute of Technology. Computational and Systems Biology Program
Format: Article
Language:English
Published: Annual Reviews 2021
Online Access:https://hdl.handle.net/1721.1/131273
Description
Summary:Induced pluripotent stem cell (iPSC) technology holds promise for modeling neurodegenerative diseases. Traditional approaches for disease modeling using animal and cellular models require knowledge of disease mutations. However, many patients with neurodegenerative diseases do not have a known genetic cause. iPSCs offer a way to generate patient-specific models and study pathways of dysfunction in an in vitro setting in order to understand the causes and subtypes of neurodegeneration. Furthermore, iPSC-based models can be used to search for candidate therapeutics using high-throughput screening. Here we review how iPSC-based models are currently being used to further our understanding of neurodegenerative diseases, as well as discuss their challenges and future directions.