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Related Concept Videos

CRISPR01:59

CRISPR

58.6K
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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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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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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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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In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Updated: Mar 11, 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

Published on: May 25, 2018

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Genome-Editing Technologies: Principles and Applications.

Thomas Gaj1, Shannon J Sirk2, Sai-Lan Shui3

  • 1Department of Bioengineering, University of California, Berkeley, California 94720.

Cold Spring Harbor Perspectives in Biology
|December 3, 2016
PubMed
Summary
This summary is machine-generated.

Genome engineering tools like CRISPR-Cas9, TALENs, and ZFNs enable precise DNA modification for disease research and gene therapy. These technologies offer powerful capabilities for manipulating genetic sequences.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Targeted nucleases allow precise manipulation of genomic sequences.
  • This capability is crucial for creating disease models and advancing gene therapy.
  • Foundational technologies include CRISPR-Cas9, TALENs, and ZFNs.

Purpose of the Study:

  • To review foundational genome engineering technologies.
  • To discuss engineering advances and achievements.
  • To explore the role of artificial transcription factors.

Main Methods:

  • Review of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9).
  • Review of transcription activator-like effector nucleases (TALENs).
  • Review of zinc-finger nucleases (ZFNs).

Main Results:

  • CRISPR-Cas9, TALENs, and ZFNs have revolutionized genome engineering.
  • Significant achievements in creating isogenic cell lines and animal models.
  • Artificial transcription factors offer complementary approaches.

Conclusions:

  • Targeted nucleases are powerful tools for biological research and therapeutic development.
  • Engineering advances continue to enhance genome editing capabilities.
  • These technologies hold immense promise for synthetic biology and human gene therapy.