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Related Concept Videos

CRISPR01:59

CRISPR

57.3K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Related Experiment Video

Updated: Dec 29, 2025

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
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Integrating CRISPR Engineering and hiPSC-Derived 2D Disease Modeling Systems.

Kristina Rehbach1,2, Michael B Fernando2,3,4, Kristen J Brennand5,2,4,6,7

  • 1Department of Genetics and Genomics.

The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|February 7, 2020
PubMed
Summary

Human induced pluripotent stem cells (hiPSCs) and CRISPR technology enable precise study of neurodegenerative and psychiatric disorders using patient-specific cells. This review guides researchers in overcoming challenges and leveraging these powerful tools for disease modeling and drug discovery.

Keywords:
CRISPRHuman induced pluripotent stem cellsdifferentiationdisease modelinginductionpsychiatric genetics

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Area of Science:

  • Neuroscience
  • Stem Cell Biology
  • Genetics

Background:

  • Human induced pluripotent stem cells (hiPSCs) offer patient-specific models for studying complex neurological and psychiatric disorders.
  • CRISPR engineering allows precise genetic modifications in hiPSCs for isogenic comparisons.
  • Existing models face challenges like cellular variability and limited maturity.

Purpose of the Study:

  • To provide a guideline for neuroscientists and clinicians transitioning to hiPSC-based research.
  • To address challenges in human in vitro disease modeling.
  • To highlight the potential of integrating hiPSC and CRISPR technologies.

Main Methods:

  • Review of state-of-the-art approaches in hiPSC differentiation and CRISPR integration.
  • Discussion of strategies to overcome interdonor/intradonor variability.
  • Overview of efficient neural induction for major brain cell types.

Main Results:

  • hiPSC and CRISPR integration enables precise isogenic comparisons of human neurons and glia.
  • Methods are presented to address limitations in neuronal maturity and circuit complexity.
  • Efficient neural differentiation strategies for key brain cell types are detailed.

Conclusions:

  • The combination of hiPSC disease modeling, CRISPR, and high-throughput methods accelerates scientific understanding.
  • This integrated approach promises significant advancements in neurodegenerative and psychiatric disorder research.
  • The field holds immense potential for future drug discovery and therapeutic development.