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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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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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What is Genetic Engineering?00:49

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Overview
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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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Related Experiment Video

Updated: Nov 20, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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Expanding the Toolbox and Targets for Gene Editing.

Jiameng Dan1, Sebastian Memczak1, Juan Carlos Izpisua Belmonte1

  • 1Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.

Trends in Molecular Medicine
|January 25, 2021
PubMed
Summary
This summary is machine-generated.

Genome editing technologies like CRISPR can modify nuclear DNA but cannot yet precisely edit mitochondrial DNA (mtDNA). This review covers advances in both nuclear and mitochondrial genome editing for genetic disease treatment.

Keywords:
gene therapygenome editingmitochondriatranslational medicine

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Genome editing offers potential for treating genetic disorders.
  • CRISPR technology enables nuclear genome modification.
  • Mitochondrial DNA (mtDNA) editing remains a significant challenge.

Purpose of the Study:

  • To review historical developments in genome editing.
  • To summarize recent advancements in nuclear genome editing.
  • To discuss progress and challenges in mitochondrial genome editing.

Main Methods:

  • Literature review of nuclear genome editing techniques.
  • Analysis of emerging mitochondrial genome editing strategies.
  • Comparative assessment of current genome editing capabilities.

Main Results:

  • CRISPR and related technologies have revolutionized nuclear genome editing.
  • Precise and efficient manipulation of mtDNA is still under development.
  • Various approaches are being explored for mtDNA editing.

Conclusions:

  • Nuclear genome editing is advancing rapidly for therapeutic applications.
  • Targeting mtDNA for genetic disease correction requires further innovation.
  • Future research will focus on developing safe and effective mtDNA editing tools.