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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

1.0K
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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CRISPR01:59

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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 and crRNAs02:53

CRISPR and crRNAs

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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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Homologous Recombination02:31

Homologous Recombination

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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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Updated: Nov 19, 2025

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells
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Emerging CRISPR/Cas9 applications for T-cell gene editing.

Roland Preece1, Christos Georgiadis1

  • 1Molecular and Cellular Immunology Unit, UCL GOS Institute of Child Health, London, U.K.

Emerging Topics in Life Sciences
|February 1, 2021
PubMed
Summary

CRISPR/Cas9 gene editing accelerates cell and gene therapies, enhancing T-cell therapies for cancer and infectious diseases. Emerging CRISPR base editing technologies offer future clinical translation potential.

Keywords:
CRISPR/Cas9T-cell receptorsbase editingchimeric antigen receptor (CAR) T cellsgenome editingimmunotherapy

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Immunotherapy

Background:

  • Gene editing tools, particularly CRISPR/Cas9, are rapidly advancing cell and gene therapy research.
  • CRISPR/Cas9 offers improved efficacy, specificity, and reduced costs, driving innovation in therapeutic applications.
  • CRISPR/Cas9 has been crucial in developing treatments for cancer, primary immunodeficiency, and infectious diseases.

Purpose of the Study:

  • To review emerging applications of CRISPR/Cas9 in T-cell therapies.
  • To address concerns regarding CRISPR-mediated indel formation.
  • To introduce CRISPR/Cas9 base editing technologies for future research and clinical translation.

Main Methods:

  • Review of current literature on CRISPR/Cas9 applications in T-cell therapy.
  • Analysis of CRISPR/Cas9 advancements, including base editing.
  • Discussion of challenges and future directions in gene editing for T-cell therapies.

Main Results:

  • CRISPR/Cas9 enhances T-cell targeting capabilities and potency for various diseases.
  • CRISPR/Cas9 base editing presents a promising avenue for precise genetic modifications.
  • Addressing indel formation is critical for safe and effective CRISPR-mediated therapies.

Conclusions:

  • CRISPR/Cas9 is a transformative technology in T-cell therapy development.
  • CRISPR base editing holds significant potential for advancing clinical translation.
  • Continued research is essential to overcome challenges and optimize gene editing strategies for therapeutic use.