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Hotta Lab
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The lab takes on gene therapy for muscular dystrophy using genome editing and iPS cells.
AboutAbout the lab
Repairing genetic mutations to treat disease
It is said that genetic mutations are involved in more than half of the intractable diseases that are difficult to treat. Technologies that repair genes and patient-derived iPS cells are powerful tools for developing new treatments for intractable diseases caused by genetic mutations.
The Hotta Lab focuses mainly on muscular dystrophy and develops a variety of technologies, including gene repair by genome editing, delivery into the body, and creation of cells that avoid immune rejection.
Key terms
- Genome editing
- A technology for cutting or rewriting DNA at targeted locations
- CRISPR-Cas9
- A representative genome editing tool that can cut DNA at a targeted location
- Muscular dystrophy
- A disease in which genetic mutations make muscles prone to damage, causing muscle strength to decline progressively
- Lipid nanoparticles
- Tiny particles made of lipids that enclose drugs or genes and deliver them to cells
ResearchResearch themes
The lab combines gene repair at the molecular level, delivery by nanoparticles, and technology at the cellular level to avoid immune rejection, aiming to develop an innovative gene therapy for muscular dystrophy.
*Illustrative imageCorrecting gene mutations with genome editing
Using patient-derived iPS cells, the lab has shown that exon skipping with CRISPR-Cas9 can repair mutated genes.
*Illustrative imageDelivering genome editing to muscle
The lab is developing NanoMEDIC, a technology that delivers CRISPR-Cas9 to muscle tissue using modified viral particles, as well as a delivery technology that uses lipid nanoparticles.
*Illustrative imageiPS cells that are less likely to be rejected by the immune system
When iPS cells from healthy donors are transplanted, differences in the HLA type on the cell surface cause immune rejection. The lab devised a method of making iPS cells in which only specific HLA genes are selectively knocked out by genome editing, greatly reducing the risk of rejection.
MembersFaculty
- Associate ProfessorAkitsu Hotta
- Program-Specific Assistant Professor (Research Base)Yusuke Kojima
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Report an errorAbout the images
The top and research theme images are illustrative and were not taken at the lab. They are cropped from Wikimedia Commons images. Cropped CC BY-SA images are provided under the same CC BY-SA license as the original (version shown on each line).
- Top image: “Cas9 5AXW” Thomas Splettstoesser (www.scistyle.com) / CC BY-SA 4.0 (cropped)
- Correcting gene mutations with genome editing: “Myoblast Fusion - Myogenesis” Darryl Leja, National Human Genome Research Institute, National Institutes of Health / Public domain (cropped)
- Delivering genome editing to muscle: “Vaccines-09-00065-g002” Michael D. Buschmann, Manuel J. Carrasco, Suman Alishetty, Mikell Paige, Mohamad G. Alameh, and Drew Weissman / CC BY 4.0 (cropped)
- iPS cells that are less likely to be rejected by the immune system: “MHC Classes” סתו כסלו / CC BY-SA 4.0 (cropped)
- Related lab (池谷研究室): “Migration of Neural Crest Cells (v2)” MWatson12 / CC BY-SA 4.0 (cropped)
- Related lab (櫻井研究室): “Skeletal muscle1-es” The original uploader was Jeremykemp at English Wikipedia. / CC BY 4.0 (cropped)
- Related lab (後藤研究室): “Bronchial anatomy” Patrick J. Lynch, medical illustrator / CC BY 2.5 (cropped)
- Related lab (齋藤研究室): “Stem Cell Research” The U.S. Food and Drug Administration / Public domain (cropped)
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