EditForce, Inc., headquartered in Fukuoka, Japan, has created the world’s first RNA-editing technology that enables RNA-editing bases to be changed from U (uracil) to C (cytosine).
The company also demonstrated that technology works in human cells in a joint study with Takahiro Nakamura, Faculty of Agriculture, Kyushu University.
The researchers said the 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.
EditForce said it will improve safety and editing efficiency in its development activities to establish innovative gene therapy technologies. The study was published in the scientific journal Communications Biology..
Outline of research
A genome existing in each human cell consists of four nucleotide bases — A (adenine), C (cytosine), G (guanine), and T (thymine). Cells generate RNA consisting of four nucleotide 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 a 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.
Genome-editing technology has been developed rapidly, but the development of editing technology for RNA sequences remains limited. To date, technologies have been established to substitute C with U, and A with G, but the substitution of other bases is yet to be realized.
Works on human cells
The study clarified the mechanism of RNA editing in plants to substitute U with C, based on which EditForce has realized the world’s first RNA-editing (base substitution) technology that substitutes U with C, and the company 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 the study opens up the possibility to edit mutations which could not have been the target with the existing technologies.
Further, repairing mutations by RNA editing without changes in genome sequence will enable EditForce to provide safer treatments to patients. The company expects that this technology will establish a novel gene therapy by improving safety and editing efficiency in its development activities.