Research groups at Kobe University, the University of Tokyo, and the University of Shizuoka have succeeded in developing a new genome editing technology "Target-AID". It solves the problem of the conventional genome editing technology that "DNA must be cut", and since it is more reliable than the conventional method, it is expected to be applied as a new gene therapy method.The results of this research were published online in "Science".
Genome editing technology, which can directly rewrite and manipulate the genes of living organisms and does not leave artificial sequences, has made remarkable progress in recent years and is being researched all over the world.Among them, genome editing technology using "artificial nuclease (enzyme artificially designed to recognize and cleave the sequence of DNA strands)" is used to obtain the target gene when the target DNA is cleaved and repaired. Since it is intended to be modified and is effective even in biological materials for which genetic manipulation is difficult, its introduction is progressing mainly in animals and plants.On the other hand, there is a problem that unexpected sequence conversion may occur in the process of DNA repair, and cells may die due to chromosomal cleavage.
The new genome editing technology developed by the research group removes nuclease activity from the conventional genome editing technology using artificial nucleases, and adds deaminase, a deamination enzyme, to construct an artificial enzyme complex.By directly reacting this with a specific part of DNA and efficiently causing a point mutation (mutation in which one base of DNA is replaced with another), we succeeded in modifying the gene function without cutting the DNA. .. It is said that the risk of DNA cleavage is extremely low, and it has been confirmed that cytotoxicity is significantly reduced compared to the conventional nuclease type.
If this technology enables efficient and intended rewriting, more sophisticated and diverse genome editing operations will be possible.In addition, since it does not place an excessive burden on cells and is highly safe, it is expected to be a technology for accelerating plant breeding and new drug development.In the future, it is also considered to be applied as a new gene therapy method for diseases such as Alzheimer's disease for which a fundamental treatment method has not been established.
