• Website
• Text in Japanese

New gene editing technologies, genetically-modified animals (mouse, rat, rabbit), and animals with human genes and tissues

Genomic editing technology (ZFN, TALEN, CRISPR / Cas9) has greatly advanced the life and medical sciences. This technology allows for the manipulation of human and animal genes for research that 1) investigates the role of a gene on cellular function, 2) creates new animal disease models, and 3) develops new treatments for human disease. We use gene editing technology to develop new tools for the modification of genes and construction of new animal models. In particular, we are using this technology to generate humanized animals, that is, animals that carry human genes. Part of this work involves growing human organs by transplanting human iPS cells in immunodeficient animals.

Rats in which GFP was knocked-in by CRISPR

1) New genome editing technology

We are developing knock-out and knock-in technology to produce genetically modified animals (mouse, rat, rabbit). We are also developing new genome editing tool to replace CRISPR.

2) Humanized animals

Animal disease models are hampered by species differences. We are looking at ways to substitute animal genes with the relevant human genes. Currently, we are conducting research on genes expressed in the liver, immune system, brain, etc.

3) Humanized animal organs

Immunodeficient animals are less likely to reject transplanted cells. Using these animals, we can grow in them human organs by transplanting human cells and tissues (liver, blood, brain, etc.).

4) New therapies from genome editing technology

Genomic editing has the potential to repair diseased genes in human cells and tissues, thus making it a potential basis for new therapies.

References

1. Yoshimi K et al. Nature Communications 7: 10431, 2016
2. Yoshimi K et al. Nature Communications 5: 4240, 2014
3. Mashimo T et al. Cell Reports 2 (3): 685-94, 2012
4. Mashimo T et al. Nature Genetics 40 (5): 514-515, 2008