EditForce, Inc., headquartered in Fukuoka, Japan, created the world’s first RNA editing technology that enables RNA editing bases from U (uracil) to C (cytosine).
The company also conducted a joint study with Takahiro Nakamura of Kyushu University’s Faculty of Agriculture, demonstrating that the technology works in human cells.
The study opens up the possibility of editing genetic mutations that cannot be targeted by existing technologies, and could potentially contribute to the development of therapies for various genetic diseases, the researchers said.
EditForce said it will improve safety and editing efficiency in its development activities to build innovative gene therapy technologies. The research is published in the scientific journal Communications Biology.
Research summary
The genome present in every human cell consists of four nucleotide bases – A (adenine), C (cytosine), G (guanine) and T (thymine). 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.
Genome editing technology has developed rapidly, but the development of RNA sequence editing technology is still limited. To date, the techniques for substituting U for C and G for A have been established, but substitution of other bases has not yet been achieved.
on human cells
This study elucidates the mechanism of plant RNA editing to replace U to C, and on this basis realizes the world’s first RNA editing (base substitution) technology to replace U to C, and proves that this technology can be used in human cells. effect.
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.
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.