Integrating CRISPR Engineering and hiPSC-Derived 2D Disease Modeling Systems
| Autor: | Kristina Rehbach, Michael B. Fernando, Kristen J. Brennand |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
Gene Editing Models Genetic Computer science General Neuroscience Induced Pluripotent Stem Cells Dual Perspectives Disease Computational biology Brain Cell 03 medical and health sciences 030104 developmental biology 0302 clinical medicine Disease risk CRISPR Humans Neural differentiation Clustered Regularly Interspaced Short Palindromic Repeats Human Induced Pluripotent Stem Cells 030217 neurology & neurosurgery |
| Zdroj: | J Neurosci |
| Popis: | Human induced pluripotent stem cells (hiPSCs) have revolutionized research on human diseases, particularly neurodegenerative and psychiatric disorders, making it possible to study mechanisms of disease risk and initiation in otherwise inaccessible patient-specific cells. Today, the integration of CRISPR engineering approaches with hiPSC-based models permits precise isogenic comparisons of human neurons and glia. This review is intended as a guideline for neuroscientists and clinicians interested in translating their research to hiPSC-based studies. It offers state-of-the-art approaches to tackling the challenges that are unique to humanin vitrodisease models, particularly interdonor and intradonor variability, and limitations in neuronal maturity and circuit complexity. Finally, we provide a detailed overview of the immense possibilities the field has to offer, highlighting efficient neural differentiation and induction strategies for the major brain cell types and providing perspective into integrating CRISPR-based methods into study design. The combination of hiPSC-based disease modeling, CRISPR technology, and high-throughput approaches promises to advance our scientific knowledge and accelerate progress in drug discovery.Dual Perspectives Companion Paper:Studying Human Neurodevelopment and Diseases Using 3D Brain Organoids, by Ai Tian, Julien Muffat, and Yun Li |
| Databáze: | OpenAIRE |
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