Department of Genome Biology

Gene Therapy Science

From the bench to the clinic, and the clinic to the bench
  • Elucidation of the cellular characteristics of cancer and development of molecular therapies
  • Analysis of regulatory mechanism of gene expression in disease onset and development
Professor Yasufumi Kaneda
Gene Therapy Science
The laboratory was founded in 1999 in response to the Graduate School of Medicine recognizing gene therapy as a new priority field. We have been promoting gene therapy research of intractable diseases based on basic biology and medicine. Through the collaboration with clinical doctors, we are also contributing to clinical applications of our research outcome.

Study of the regulatory mechanisms for gene expressions associated with cancer and congenital heart disease

The lab has developed a new drug delivery system (DDS), the HVJ envelope vector (HVJ-E), which is based on an inactive Sendai virus vector and can deliver genes and siRNA to various cells both in vitro and in vivo. HVJ-E has been shown to induce apoptosis in cancer cells as well as stimulate anti-tumor activity in immune cells. This DDS is currently being developed for clinical application. To understand how HVJ-E promotes apoptosis in cancer, the lab is using an assortment of technologies, including CRISPR/Cas9 genome editing, next generation sequencers, epigenetics, and molecular imaging. Finally, we are exploring genes essential for cancer stem cells and responsible for chemotherapy-resistance.

In research regarding congenital heart disease, we have identified the cooperative roles of transcription factors and histone modifying enzymes. Based on our findings related to a network of transcription factors and RNA metabolism, we will be able to elucidate new insights on the onset of congenital heart disease.