Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

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...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Toll-like receptor 5 protects against murine lung fibrosis through reduced dysbiosis, and <i>TLR5</i> deficiency is associated with human IPF.

Science translational medicine·2026

Same author

Maternal postpartum sedentary life exacerbates the metabolic impact of moderately fat-rich diet in male mice.

npj metabolic health and disease·2025

Same author

High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa.

Molecular therapy. Methods & clinical development·2025

Same author

Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia.

JCI insight·2025

Same author

CRISPR/Cas9 gene therapy increases the risk of tumorigenesis in the mouse model of hereditary tyrosinemia type I.

JHEP reports : innovation in hepatology·2025

Same author

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.

Science translational medicine·2025

Search research articles

Related Experiment Video

Updated: Dec 28, 2025

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Published on: June 2, 2022

Using CRISPR/Cas9 to model human liver disease.

Michele Alves-Bezerra^1,2, Nika Furey^1,2,3, Collin G Johnson^1,2

¹Center for Cell and Gene Therapy, Stem Cells and Regenerative Medicine Center, Baylor College of Medicine, Houston, TX, USA.

JHEP Reports : Innovation in Hepatology

|February 11, 2020

Summary

CRISPR gene editing technology enables the creation of advanced animal models for studying human liver diseases. These models improve our understanding of disease mechanisms and aid in developing new treatments.

More Related Videos

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Published on: May 19, 2018

Related Experiment Videos

Last Updated: Dec 28, 2025

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Published on: June 2, 2022

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Published on: May 19, 2018

Area of Science:

Biomedical Research
Genetics
Animal Models

Background:

CRISPR/Cas9 gene editing has transformed biomedical research.
Its ease of design facilitates disease modeling in various animal species.
The liver is a target for somatic genome editing, with efficient whole-liver editing methods emerging.

Purpose of the Study:

To review CRISPR-edited animal models for human liver disorders.
To cover acquired and inherited hepatic metabolic diseases and liver cancers.
To highlight advancements in understanding liver disease pathophysiology.

Main Methods:

Utilizing CRISPR/Cas9 technology for gene editing in animals.
Developing efficient delivery methods for somatic genome editing in the liver.
Generating diverse animal models for specific human liver conditions.

Main Results:

CRISPR enables high-efficiency gene editing in multiple species beyond mice.
Development of animal models for a wide spectrum of liver diseases.
Enhanced ability to study liver disease pathophysiology through advanced models.

Conclusions:

CRISPR technology has significantly expanded the availability of animal models for human liver diseases.
These models are crucial for advancing the understanding of liver disease.
CRISPR-edited animals offer new avenues for developing innovative therapeutic strategies for liver disorders.