Abstract

Stem and derivative cells induced from somatic tissues are a critical tool for disease modeling but significant technical hurdles hamper their use. The purpose of this review is to provide an overview of pitfalls and mitigation strategies for the nonstem cell biologist using induced pluripotent stem cells and investigating neurodevelopmental disorders. What sample sizes are reasonable? What derivation and purification protocols should be used to make human neurons? In what way should gene editing technologies be used to support discoveries? What kinds of preclinical studies are the most feasible? It is hoped that this roadmap will provide the necessary details for experimental planning and execution for those less familiar in the area of stem cell disease modeling. High‐quality human preclinical models will allow for the discovery of molecular and cellular phenotypes specific to different neurodevelopmental disorders, and may provide the assays to advance translational medicine for unmet medical needs.

Details

Title
A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
Author
Ernst, Carl 1   VIAFID ORCID Logo 

1  Department of Human Genetics, McGill University and Douglas Hospital Research Institute, Montreal, Quebec, Canada; Department of Psychiatry, McGill University and Douglas Hospital Research Institute, Montreal, Quebec, Canada; Department of Neurology and Neurosurgery, McGill University and Douglas Hospital Research Institute, Montreal, Quebec, Canada 
Pages
567-574
Section
CONCISE REVIEWS
Publication year
2020
Publication date
May 2020
Publisher
Oxford University Press
ISSN
21576564
e-ISSN
21576580
Source type
Scholarly Journal
Language of publication
English
DOI
https://doi.org/10.1002/sctm.19-0344
ProQuest document ID
2393947686
Copyright
© 2020. This work is published under http://creativecommons.org/licenses/by-nc/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.