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Related Concept Videos

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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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

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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.
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Homologous Recombination02:31

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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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Conservative Site-specific Recombination and Phase Variation02:53

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
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Updated: Mar 7, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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Precision genome editing in the CRISPR era.

Jayme Salsman1, Graham Dellaire1,2,3

  • 1a Department of Pathology, Dalhousie University, Halifax, NS B3H 4R2, Canada.

Biochemistry and Cell Biology = Biochimie Et Biologie Cellulaire
|February 9, 2017
PubMed
Summary

CRISPR-Cas9 technology enables precise genome editing for gene therapy. Enhancing homology-directed repair (HDR) with CRISPR-Cas9 improves gene editing accuracy for therapeutic applications.

Keywords:
CRISPRCas9DNA repairgene therapygenome editingréparation d’ADNthérapie géniqueédition génique

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR-Cas9 technology allows targeted DNA double-strand breaks (DSBs) at specific genomic loci.
  • DSB repair occurs via non-homologous end-joining (NHEJ) for gene disruption or homology-directed repair (HDR) for precise editing.
  • Current research focuses on improving CRISPR-Cas9 fidelity and HDR efficiency for gene therapy.

Purpose of the Study:

  • To review CRISPR-Cas9 applications for gene inactivation and genome editing.
  • To focus on methods for enhancing CRISPR-Cas9-mediated HDR.
  • To discuss the generation of cell and animal models using enhanced HDR.

Main Methods:

  • Review of scientific literature on CRISPR-Cas9 technology and gene editing.
  • Analysis of approaches to increase homology-directed repair (HDR) efficiency.
  • Discussion of CRISPR-Cas9 applications in creating genetic models.

Main Results:

  • CRISPR-Cas9 facilitates precise gene editing through HDR, enabling gene knockouts, point mutations, and sequence insertions.
  • Strategies to enhance HDR efficiency are crucial for precise genome engineering.
  • CRISPR-Cas9-mediated HDR is instrumental in generating advanced cell and animal models.

Conclusions:

  • CRISPR-Cas9 technology has revolutionized genetic engineering and holds significant promise for gene therapy.
  • Enhancing HDR efficiency is key to unlocking the full potential of CRISPR-Cas9 for precise human somatic cell gene therapy.
  • Further research is needed to address challenges and advance CRISPR-Cas9 applications in clinical settings.