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A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists

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...

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Detalles Bibliográficos
Autor principal: Ernst, Carl
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7180294/
https://www.ncbi.nlm.nih.gov/pubmed/32052596
http://dx.doi.org/10.1002/sctm.19-0344
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author Ernst, Carl
author_facet Ernst, Carl
author_sort Ernst, Carl
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description 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.
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spelling pubmed-71802942020-04-27 A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists Ernst, Carl Stem Cells Transl Med Concise Reviews 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. John Wiley & Sons, Inc. 2020-02-13 /pmc/articles/PMC7180294/ /pubmed/32052596 http://dx.doi.org/10.1002/sctm.19-0344 Text en © 2020 The Author. stem cells translational medicine published by Wiley Periodicals, Inc. on behalf of AlphaMed Press This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
spellingShingle Concise Reviews
Ernst, Carl
A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title_full A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title_fullStr A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title_full_unstemmed A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title_short A roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
title_sort roadmap for neurodevelopmental disease modeling for non‐stem cell biologists
topic Concise Reviews
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7180294/
https://www.ncbi.nlm.nih.gov/pubmed/32052596
http://dx.doi.org/10.1002/sctm.19-0344
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