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In Vivo Modeling of Skeletal Muscle Diseases Using the CRISPR/Cas9 System in Rats
Katsuyuki Nakamura1, Takao Tanaka2, Keitaro Yamanouchi3
1Department of Veterinary Physiology, Graduate School of Agricultural and Life Sciences, The University of Tokyo, Tokyo, Japan.
Methods in Molecular Biology (Clifton, N.J.)
|March 30, 2023
Summary
Gene editing with CRISPR/Cas9 enables creating Dystrophin mutant rats, which exhibit more severe phenotypes than mice. These rats serve as a superior model for studying human Duchenne muscular dystrophy.
Area of Science:
- Molecular Biology
- Genetics
- Animal Models
Background:
- The CRISPR/Cas9 system is a versatile gene editing technology applicable across species.
- Duchenne muscular dystrophy (DMD) in humans is linked to the Dystrophin gene.
- Existing Dystrophin mutant mouse models do not fully replicate severe human DMD phenotypes.
Purpose of the Study:
- To detail a protocol for generating gene-modified rats using CRISPR/Cas9.
- To establish a more accurate animal model for human Duchenne muscular dystrophy.
Main Methods:
- Utilized the CRISPR/Cas9 gene editing system.
- Employed microinjection into rat embryos for genetic modification.
- Generated Dystrophin gene mutant rats.
Main Results:
- Dystrophin mutant rats displayed more severe muscle degenerating phenotypes compared to mice.
- The observed phenotypes in rats closely resemble the characteristics of human DMD.
- Rats demonstrate greater utility as models for human skeletal muscle diseases.
Conclusions:
- CRISPR/Cas9 facilitates the creation of relevant Dystrophin mutant rat models.
- Rats offer a more representative preclinical model for Duchenne muscular dystrophy than mice.
- This protocol enables the generation of gene-modified rats for disease modeling.
