Fukuoka, Japan, October 18 January 2022 /PRNewswire/ — EditForce, Inc. (“EditForce”), headquartered in Fukuoka, Japanhas achieved the world’s first RNA editing technology to change RNA editing bases from “U” to “C”, and demonstrated in a joint study with Prof. that the technology can even work in human cells Takahiro Nakamuraagricultural college, Kyushu UniversityThis research opens up the possibility of editing genetic mutations that cannot be targeted by existing technologies and is expected to contribute to the development of treatments for various genetic diseases. EditForce will improve safety and editing efficiency in its development activities to build innovative gene therapy technologies.The study was published in the scientific journal Communications Biology September 15 (September 16 in JST), 2022.
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Research summary
The genome, which exists in every human cell, consists of four nucleotide bases—A, C, G, and T—in which sequences contain the information needed to make up the human body. Cells produce RNA, which consists of the four nucleotide bases A, C, G, and U, based on the sequence of the genome, and then produce proteins based on the sequence of the RNA. However, single nucleotide changes in the genome or RNA sequence can cause a variety of diseases. Treating these diseases requires single-base editing techniques or repairing genomic or RNA mutations to get the sequence back on track.
At present, genome editing technology has developed rapidly, but the development of RNA sequence editing technology is still limited. In particular, with respect to single base substitution, techniques for replacing “C” with “U” and “A” with “G” have been established, but other base substitutions have not yet been realized.
This study elucidates the mechanism by which RNA editing replaces “U” with “C” in plants, and on this basis realizes the world’s first RNA editing (base replacement) technology that replaces “U” with “C”, Inc. Demonstrate that the technique works in human cells.
Base editing techniques can be used to treat diseases caused by a single mutation, and the study’s “U-to-C” RNA editing technique opens up the possibility of editing mutations that cannot be targeted by existing techniques. technology. In addition, repairing mutations through RNA editing without altering the genome sequence will enable EditForce to provide patients with safer treatments. The company expects that the technology will establish a new gene therapy class by improving the safety and editing efficiency of its development activities.
This work was supported by the Young Scholars Research Fund funded by Yamanashi Prefecture.
Article information
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 synthesis of PPR-DYW protein Bacteria and Human Cultured Cells” Commun Biol. 5, 968 (2022).
About EditForce, Inc.
EditForce, Inc., a Kyushu University– Founding venture company to develop unique DNA/RNA editing technology (PPR platform technology) ),was founded in May 2015 at KISCO Ltd. and Prof. Takahiro Nakamura
Kyushu University (Former President of EditForce, current Scientific Advisor), funded by companies and funds with a track record of investing in life sciences and biotechnology. EditForce aims to discover medicines using PPR technology through joint research with universities and private companies.
For more information, please visit: https://www.editforce.co.jp/
Pentapeptide Repeat (PPR) Protein Platform TechnologyPPR is a protein found in plants that regulates gene expression by binding to DNA and RNA in a sequence-specific manner. PPR proteins are also present in humans and yeast, which have similar functions. Professor Takahiro Nakamura and Dr.
Yagi Yusuke
Chief Technology Officer at EditForce, focused on PPR proteins and elucidated the mechanisms that determine sequence specificity, and established a technology for creating a variety of PPR proteins, each of which binds to a specific target DNA or RNA sequence. In addition, target genomes and RNAs inside and outside the cell can be manipulated and modified by fusion with effector proteins. SourceEditForce, Inc.
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