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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...

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...

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Related Experiment Video

Updated: Nov 8, 2025

Author Spotlight: Advancing Techniques and Discoveries in Protein Synthesis and Assembly Through Innovative Mitochondrial Research

Author Spotlight: Advancing Techniques and Discoveries in Protein Synthesis and Assembly Through Innovative Mitochondrial Research

Published on: June 7, 2024

Adapting CRISPR/Cas9 System for Targeting Mitochondrial Genome.

Syed-Rehan A Hussain^1,2, Mehmet E Yalvac³, Benedict Khoo¹

¹Center for Molecular and Human Genetics, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, United States.

Frontiers in Genetics

|April 23, 2021

Summary

This study demonstrates a novel CRISPR-Cas9 method for mitochondrial gene editing. The approach successfully targets mitochondrial DNA, reducing expression of a specific gene variant and offering potential for treating mitochondrial diseases.

Keywords:

PNPase RP-loop chimeric guide RNA heteroplasmic mutations mitochondria

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CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

Published on: March 12, 2018

Area of Science:

Molecular Biology
Genetics
Biotechnology

Background:

Mitochondrial gene editing is challenging due to inefficient delivery of CRISPR-Cas9 components.
Targeting mitochondrial DNA (mtDNA) is crucial for treating various inherited diseases.

Purpose of the Study:

To develop and validate a novel CRISPR-Cas9 delivery system for effective mitochondrial gene editing.
To demonstrate sequence-specific cleavage of mitochondrial DNA and reduction of pathogenic gene expression.

Main Methods:

Engineered a guide RNA (gRNA) with an RNA transport-derived stem loop element (RP-loop) for mitochondrial targeting.
Expressed Cas9 enzyme fused with a mitochondrial localization sequence.
Utilized cells with a specific 11205G variant in the NADH-ubiquinone oxidoreductase chain 4 (ND4) gene.

Main Results:

Achieved mitochondrial colocalization of the RP-loop gRNA.
Observed a significant reduction in ND4 gene expression in cells with the targeted variant.
Reported a decrease in overall mitochondrial DNA (mtDNA) levels following gene editing.

Conclusions:

The developed RP-loop gRNA system facilitates mitochondrial delivery and function of CRISPR-Cas9.
This proof-of-concept study validates sequence-specific mtDNA cleavage and gene expression reduction.
The findings support the potential of CRISPR-Cas9-mediated gene editing for treating mitochondrial diseases.