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Related Concept Videos

CRISPR01:59

CRISPR

56.9K
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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Genetic Screens02:46

Genetic Screens

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Genetic screens are tools used to identify genes and mutations responsible for phenotypes of interest. Genetic screens help identify individuals or a group of people at risk of developing  genetic diseases and help them with early intervention, targeted therapy, and reproductive options.
Forward genetic screens
Forward or “classical” genetic screens involve creating random mutations in an organism’s DNA using radiation, mutagens, or insertion of additional bases, which...
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Related Experiment Video

Updated: Dec 15, 2025

CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer
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CRISPR-based functional genomics for neurological disease.

Martin Kampmann1,2,3

  • 1Institute for Neurodegenerative Diseases, University of California, San Francisco, CA, USA. martin.kampmann@ucsf.edu.

Nature Reviews. Neurology
|July 10, 2020
PubMed
Summary
This summary is machine-generated.

CRISPR functional genomics screens, combined with stem cell technology, are revolutionizing the study of neurological diseases. These powerful tools help uncover disease mechanisms and identify potential therapeutic targets for conditions like neurodegenerative disorders.

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A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
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A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

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A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

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Area of Science:

  • Genomics
  • Neuroscience
  • Biotechnology

Background:

  • Neurological disorders pose significant public health challenges due to a lack of effective treatments.
  • Understanding the molecular and cellular mechanisms underlying these diseases is crucial for developing therapies.
  • Genome-wide association studies (GWAS) identify genetic variants linked to diseases, but their functional impact remains largely unknown.

Purpose of the Study:

  • To review how CRISPR-based functional genomics approaches can elucidate disease mechanisms in neurological disorders.
  • To highlight the potential of CRISPR technologies in identifying novel therapeutic targets.
  • To discuss the integration of CRISPR screens with induced pluripotent stem cell (iPSC) technology for disease modeling.

Main Methods:

  • Utilizing CRISPR interference (CRISPRi) and CRISPR activation (CRISPRa) for genome editing and gene expression control in experimental models.
  • Implementing massively parallel genetic screens to assess the functional consequences of genetic perturbations in human cells.
  • Combining CRISPR screens with iPSC technology to generate patient-derived neurons, glia, and brain organoids for disease modeling.

Main Results:

  • CRISPR screens enable the evaluation of functional consequences of genetic variations in cellular models.
  • CRISPRi and CRISPRa can model disease-associated gene expression changes to pinpoint causal factors.
  • Genetic modifier screens identify key determinants of cell-type-specific vulnerability and potential therapeutic targets.

Conclusions:

  • CRISPR-based functional genomics offers powerful strategies to uncover disease mechanisms in neurological conditions.
  • The integration of CRISPR technology with iPSC-derived models provides unprecedented insights into human neurological diseases.
  • These approaches are pivotal for advancing the development of much-needed disease-modifying treatments for neurological disorders.