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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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Conservative Site-specific Recombination and Phase Variation

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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CRISPR and crRNAs02:53

CRISPR and crRNAs

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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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 Short...
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Homologous Recombination

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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Related Experiment Video

Updated: Jun 5, 2026

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
09:51

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation

Published on: February 2, 2016

A TALE nuclease architecture for efficient genome editing.

Jeffrey C Miller1, Siyuan Tan, Guijuan Qiao

  • 1Sangamo BioSciences, Inc., Richmond, California, USA.

Nature Biotechnology
|December 24, 2010
PubMed
Summary

Scientists engineered novel DNA-cutting enzymes called transcription activator-like effector (TALE) nucleases. These engineered TALE nucleases precisely modify genes in human cells, offering new tools for gene editing and regulation.

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Related Experiment Videos

Last Updated: Jun 5, 2026

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
09:51

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation

Published on: February 2, 2016

Mouse Genome Engineering Using Designer Nucleases
12:04

Mouse Genome Engineering Using Designer Nucleases

Published on: April 2, 2014

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Targeted genome modification requires precise DNA-cleaving tools.
  • Transcription activator-like effectors (TALEs) offer customizable DNA-binding specificity.

Purpose of the Study:

  • To develop novel engineered nucleases based on TALE proteins for targeted gene editing.
  • To demonstrate the application of TALE nucleases for modifying endogenous genes in human cells.

Main Methods:

  • Designing TALE truncation variants fused to the FokI catalytic domain.
  • Introducing these engineered TALE nucleases into human cells.
  • Assessing gene editing efficiency at endogenous NTF3 and CCR5 loci.

Main Results:

  • Identified TALE truncation variants that function as active nucleases.
  • Achieved targeted gene editing, including discrete edits and small deletions, with efficiencies up to 25%.
  • Demonstrated regulation of endogenous mammalian genes using designed TALE transcription factors.

Conclusions:

  • Designed TALE nucleases are effective tools for targeted genome modification in human cells.
  • This strategy enables precise regulation and editing of endogenous genes for research and therapeutic applications.