EditForce Realizes RNA-editing Technology of “U-to-C” in Human Cells
EditForce, Inc. (President and CEO: Takashi Ono; hereinafter “EditForce”) has realized the world’s first RNA-editing technology that enables editing RNA bases from “U” to “C”, and demonstrated that this technology works even in human cells in the joint study with Professor Takahiro Nakamura, Faculty of Agriculture, Kyushu University.
This study opens up the possibility of editing gene mutations which could not have been the target with the existing technologies, and is expected to contribute to the R&D of therapies for various genetic diseases. We will improve safety and editing efficiency in our development activities to establish innovative gene therapy technologies.
This study was published in the scientific journal "Communications Biology" on September 15 (September 16 in JST).
Outline of Research
A genome existing in each of our cells consist of four nucleotide bases-A, C, G, and T of which the sequence holds the information necessary to make up a human body. Cells generate RNA consisting of four nucleotides bases- A, C, G, and U, based on genomic sequences, and then produce protein according to the sequences of RNA. However, changes in a single nucleotide on genome or RNA sequence cause a variety of diseases. Treatment of these diseases requires single base editing technology of repairing genomic or RNA mutations to bring the sequences back on the normal track.
Currently, genome editing technology has been developed rapidly, but the development of editing technology for RNA sequence remains limited. Especially as regards a single base substitution, although technologies have been established to substitute "C" with "U" and "A" with "G", the substitution of other bases is to be realized.
This study clarified the mechanism of RNA editing in plants to substitute “U” with “C”, based on which we have realized the world's first RNA editing (base substitution) technology which substitutes "U" with "C" and we demonstrated that this technology works in human cells.
Base editing technologies can be applied to the treatment of diseases caused by a single mutation, and "U-to-C" RNA editing technology of this study opens up the possibilities to edit mutations which could not have been the target with the existing technologies. Further, repairing mutations in RNA, without manipulation of genome sequence, will enable us to provide safer treatments to patients. We expect that this technology will establish a novel gene therapy by improving safety and editing efficiency in the development activities.
Ichinose M., Kawabata M., Akaiwa Y., Shimajiri Y., Nakamura I., Tamai T., Nakamura T., Yagi Y. and Gutmann B. U-to-C RNA editing by synthetic PPR-DYW proteins in bacteria and human culture cellsArchives