News Releases & Research Results Efficient gene suppression with a DNA/DNA double-stranded oligonucleotide as a candidate for novel nucleic acid drugs - Advances toward the development of therapeutics for intractable disease -

The results of collaborative research and development conducted by Professor Takanori Yokota, Project Associate Professor Tetsuya Nagata, Specially Appointed Assistant Professor Kotaro Yoshioka, and Graduate student Yutarou Asami of the Department of Neurology and Neurological Science (Department of Neurology), Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University and Ionis Pharmaceuticals, Inc.

• A novel technology was developed to dramatically improve the effects of conventional RNA-targeting antisense nucleic acid drugs.
• Specifically, an innovative next-generation nucleic acid drug, “DNA/DNA double-stranded oligonucleotide,” was developed based on the previously developed next-generation nucleic acid drug, “DNA/RNA heteroduplex oligonucleotide.”
• The DNA/DNA double-stranded oligonucleotide, produced through this research and development, is a basic technology applicable to RNA-targeting therapeutics for various diseases and will be applied to intractable diseases, such as hepatitis B, non-alcoholic steatohepatitis, and Alzheimer's disease, for which there has been no effective treatment.

This project was conducted with the support of the Science and Technology Platform Program for Advanced Biological Medicine by AMED.

The results were published online in the international scientific journal Molecular Therapy on December 8.

Asami Y., et al. Efficient Gene Suppression by DNA/DNA Double-Stranded Oligonucleotide In Vivo Molecular Therapy
DOI: 10.1016/j.ymthe.2020.10.017