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CRISPR

CRISPR

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...

CRISPR/Cas9 Genome Editing

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...

What is Genetic Engineering?

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Conservative Site-specific Recombination and Phase Variation

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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In-vitro Mutagenesis

In-vitro Mutagenesis

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.

CRISPR and crRNAs

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.
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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Related Experiment Video

Updated: Mar 9, 2026

Mouse Genome Engineering Using Designer Nucleases

Mouse Genome Engineering Using Designer Nucleases

Published on: April 2, 2014

Therapeutic genome editing with engineered nucleases.

Simone A Haas, Viviane Dettmer, Toni Cathomen¹

¹Toni Cathomen, Ph.D., Institute for Cell and Gene Therapy, Medical Center - University of Freiburg, Hugstetter Str. 55, 79106 Freiburg, Germany, Phone: +49 761 270 34800, Fax: + 49 761 270 37900,

Hamostaseologie

|January 11, 2017

Summary

This summary is machine-generated.

Designer nucleases like CRISPR-Cas offer precise genome surgery for genetic disorders previously untreatable by gene therapy. These engineered nucleases also hold promise for infectious diseases and cancer immunotherapies.

Keywords:

clinical application clinical trial designer nuclease gene editing gene therapy genetic disorder

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Last Updated: Mar 9, 2026

Mouse Genome Engineering Using Designer Nucleases

Mouse Genome Engineering Using Designer Nucleases

Published on: April 2, 2014

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

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

Published on: February 2, 2016

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Area of Science:

Molecular Biology
Genetics
Biotechnology

Background:

Gene therapy has advanced with the development of programmable nucleases.
Designer nucleases include zinc finger nucleases, TALE nucleases, and CRISPR-Cas systems.
These tools enable precise genome editing, offering new therapeutic avenues.

Purpose of the Study:

To review the development of programmable nucleases.
To discuss challenges and improvements in clinical translation of gene editing.
To provide an outlook on future clinical applications.

Main Methods:

Review of scientific literature on designer nucleases.
Analysis of gene editing technologies and their clinical potential.
Discussion of challenges in translating gene editing to clinical practice.

Main Results:

Programmable nucleases enable precise genome surgery for genetic disorders.
Engineered nucleases offer novel strategies for infectious diseases and cancer immunotherapy.
Significant progress has been made in translating gene editing into clinical use.

Conclusions:

Designer nucleases represent a significant advancement in gene therapy.
Clinical applications of gene editing are expanding, with future potential in various diseases.
Further research and development are crucial for realizing the full potential of gene editing therapies.