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Related Concept Videos

CRISPR01:59

CRISPR

52.9K
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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CRISPR and crRNAs02:53

CRISPR and crRNAs

17.4K
Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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Related Experiment Video

Updated: Sep 13, 2025

Efficient Production and Identification of CRISPR/Cas9-generated Gene Knockouts in the Model System Danio rerio
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Efficient Production and Identification of CRISPR/Cas9-generated Gene Knockouts in the Model System Danio rerio

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CRISPR-based functional genomics tools in vertebrate models.

Gaurav K Varshney1, Shawn M Burgess2

  • 1Genes & Human Disease Research Program, Oklahoma Medical Research Foundation, Oklahoma City, OK, USA. Gaurav-varshney@omrf.org.

Experimental & Molecular Medicine
|July 31, 2025
PubMed
Summary
This summary is machine-generated.

CRISPR-Cas technologies revolutionize functional genomics in vertebrate models. These advanced gene editing tools, including base and prime editors, accelerate gene function discovery and disease modeling for potential therapies.

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Area of Science:

  • Genomics
  • Molecular Biology
  • Biotechnology

Background:

  • CRISPR-Cas technologies have transformed genetic manipulation in research.
  • CRISPR applications are widespread in vertebrate models like mice and zebrafish.
  • Advancements include high-throughput screening, knock-in alleles, and large-scale screens.

Purpose of the Study:

  • To review the impact of CRISPR-based tools in vertebrate models.
  • To highlight the utility of CRISPR in functional genomics and disease modeling.
  • To discuss innovative CRISPR technologies and their applications.

Main Methods:

  • Adaptation of CRISPR-Cas for high-throughput mutagenesis.
  • Development of base editors for single-nucleotide modifications.
  • Implementation of prime editors for precise edits without double-strand breaks.
  • Utilizing CRISPR interference and activation for transcriptional modulation.
  • Employing newer methods like MIC-Drop and Perturb-seq for in vivo screening.

Main Results:

  • CRISPR tools enable precise genetic manipulations in vertebrate models.
  • CRISPR facilitates understanding of gene functions in development, physiology, and pathology.
  • Innovative CRISPR technologies expand the toolkit for genetic research.
  • CRISPR-based gene therapies show promise for clinical translation, including for sickle cell disease.

Conclusions:

  • CRISPR-based tools have a transformative impact on vertebrate functional genomics.
  • These technologies are crucial for advancing disease modeling and understanding complex biological processes.
  • The ongoing development of CRISPR methods promises further breakthroughs in research and therapeutics.