Molecules that compose cells = Targets for drug discovery
Our bodies are an aggregate of numerous cells. The behavior of individual cells is determined by the functions of DNA (genome), RNA, and proteins and the relationships between them, which are complex. It is known that abnormalities in cell behavior lead to diseases. A variety of drugs, such as antibody technologies, are currently being discovered and developed. Antibodies directly bind to the proteins responsible for a relevant disease and inhibit their functions. However, it is difficult to design binding molecules because proteins have a combination of 20 amino acids and have a complex steric structure. On the contrary, DNA and RNA, which are defined by the sequence of four bases (i.e., adenine [A], cytosine [C], thymine [T], and guanine [G]), have less combinations and are less complex than proteins.
We possess technology that can construct molecules that specifically bind to these four bases. By taking advantage of this technology, we can create new molecules that target the base sequences of DNA and RNA. Currently, we are using such molecules for drug discovery and development.
PPR Technology × RNA Editing = NEW GENE & CELL THERAPY!!
Artificial molecules that can freely manipulate various nucleic acid molecules in the cells can be prepared by catalyzing PPR proteins that bind to the target and nucleic acids or by fusing proteins that control gene expression.
For example, various causative RNA molecules and gene expression pathways that are associated with diseases are known. Our technologies allow for the design of a PPR that makes those abnormal RNAs return to normal or eliminate them. We think that the induction of the in vivo expression of the PPR protein via a viral vector, direct protein introduction, or other means will lead to treatment.
Overview of Related Technologies
The RNA-PPR technology is the only RNA-binding protein with high design flexibility.
The strengths of protein molecules are as follows:
1. It is possible to develop various nucleic acid–control tools because they can be fused with functionmolecules relatively easily.
2. It is possible to optimize binding with nucleic acids by using protein engineering.
Our development takes advantage of these characteristics.
For DNA-binding molecules, various types of technologies have been developed, thus accelerating advances in genome-editing technologies. Furthermore, our DNA-PPR technology is a genome-editing technology developed in Japan and has a mechanism that is different from those of other technologies.