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Molecular Genetic Medicine Lab
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The lab develops gene therapies and cell therapies using viral vector and stem cell technologies.
AboutAbout the lab
Building therapies with viral vectors and stem cells
The lab researches the development of the foundational technologies for gene therapy and cell therapy.
Building on viral vector and tissue stem cell technologies, the lab advances research in gene therapy and cell therapy.
Alongside analysis of molecular pathology and clinical genetics services, the lab aims to conduct translational research for personalized genomic medicine.
The lab is a department based in the Center for Gene and Cell Therapy at the Institute of Medical Science Hospital; the center handles research, development, and clinical trials of treatments for intractable cancers and genetic diseases.
Key terms
- Viral vector
- A virus modified so that it does not cause disease, used as a tool to deliver genes into cells
- AAV vector
- A viral vector made from adeno-associated virus. It is considered to be highly safe and to work for a long time
- Translational research
- Research that aims to connect findings from basic research to actual diagnosis and treatment
ResearchResearch themes
Centered on developing foundational technologies for gene therapy and cell therapy, the lab also works on analysis of molecular pathology, clinical genetics, and the refinement of therapies. By combining viral vector and stem cell technologies, it works to create treatments for intractable diseases.
*Illustrative imageDeveloping viral vector technologies
The lab develops technologies to bring AAV vectors, which are expected to offer high safety and long-lasting expression, into practical use as products for gene therapy. Its work includes developing new variants, establishing manufacturing and purification processes, and evaluating quality using analytical ultracentrifugation and cryo-electron microscopy.
*Illustrative imageDeveloping therapies for neuromuscular diseases
The lab is developing treatments for Duchenne muscular dystrophy, for which there is no fundamental cure. Using disease model animals such as mice and dogs, as well as primates, it tests the safety and efficacy of miniaturized dystrophin and of methods to sustain gene expression over a long period.
*Illustrative imageTherapies using somatic stem cells
Depending on the disease state and genetic predisposition, stem cell therapy, or a combination of gene therapy and stem cell therapy, may be the more suitable approach. For muscular dystrophy and acute-phase cerebral infarction, the lab has confirmed safety and efficacy in disease model animals.
*Illustrative imageBiomarker discovery
The lab searches for the biomarkers needed to assess disease status in clinical trials. Through comprehensive analyses using Duchenne muscular dystrophy model animals, it has identified many candidate substances.
MembersFaculty
The lab's members consist of faculty, project researchers, visiting lecturers, collaborative researchers, technical specialists, dispatched researchers, academic specialists, graduate students, and research trainees.
- ProfessorTakashi OkadaPublications ↗
- Project Associate ProfessorYasushi SodaPublications ↗
- Project LecturerYuko KasaharaPublications ↗
- Assistant ProfessorYuji TsunekawaPublications ↗
- Project Assistant ProfessorHiromi KinohPublications ↗
- Project Assistant ProfessorKen Sugo
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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: “Human Stem Cells ( )” National Center for Advancing Translational Sciences from Bethesda, MD / Public domain (cropped)
- Developing viral vector technologies: “Beckman Coulter Ultracentrifuge” Karbohut / CC BY-SA 4.0 (cropped)
- Developing therapies for neuromuscular diseases: “Skeletal muscle 400x” Alexander G. Cheroske / CC BY-SA 4.0 (cropped)
- Therapies using somatic stem cells: “Dental pulp stem cells. SEM-BSE” Ivan A. Novikov / CC BY-SA 4.0 (cropped)
- Biomarker discovery: “378th EMDS hosts Walking Blood Bank Pre-screening (9006795)” U.S. Air Force photo by Senior Airman Kevin Dunkleberger / Public domain (cropped)
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