Gene editing technology is a future oriented technology, represented by CRISPR, which has basically achieved "single modification" of genes - precise editing at the single base and short sequence scales. So, can a new gene editing technology be invented to achieve comprehensive coverage in one modification? Biologists from the Institute of Zoology, Chinese Academy of Sciences/Beijing Institute of Stem Cell and Regenerative Medicine have developed a new technology of gene editing with independent intellectual property rights, successfully achieved precise gene writing using ribonucleic acid (RNA) as the medium, and provided a basis for the development of a new generation of innovative gene therapy. This achievement was jointly completed by Li Wei, a researcher from the Institute of Zoology, Chinese Academy of Sciences/Beijing Institute of Stem Cell and Regenerative Medicine, and Zhou Qi, a researcher team. The related papers were published on the international academic journal Cell published on the evening of July 8. Li Wei introduced that genomic deoxyribonucleic acid (DNA) is the blueprint of life, and precise manipulation of genomic DNA at any scale represents the underlying ability to modify and draw the blueprint of life, which is the core of the development of genetic engineering technology. At present, achieving efficient and precise integration of large-scale DNA fragments into the genome is an urgent challenge that needs to be overcome in the entire field of genetic engineering. In response to this major technological challenge, various gene writing techniques have been developed, but most of these techniques rely on DNA templates as donors for gene writing. In practical medical applications, DNA donors face many challenges such as high immunogenicity, difficult in vivo delivery, and the risk of random integration in the genome. The researchers turned their focus to RNA donors. RNA donors have the characteristics of lower immunogenicity, effective delivery by non viral vectors, rapid degradation within cells, and no risk of random integration. Large fragment precise writing technology using RNA as a donor has significant advantages in safety and delivery. After multiple attempts, the research team selected the R2 retrotransposon for research. Li Wei introduced, "By combining genomic data mining and macromolecular engineering, we have developed an R2 retrotransposon tool that uses RNA donors for precise writing of large fragment genes. It can achieve efficient and accurate integration of large fragment genes in various mammalian cell lines and primary cells, with a maximum efficiency of over 60%." This breakthrough in technology means that precise writing of exogenous functional genes can be used to intervene in genetic defects and other diseases caused by genes covering multiple mutation profiles at different sites, and can develop more universal gene and cell therapies with broad application prospects. Li Wei said, "Currently, this technology cannot achieve programmable writing at different genomic loci, and the gene writing efficiency in human primary cells is relatively low. Therefore, further development and optimization are needed in the future. This is also the focus of our next work
Edit：Xiong Dafei Responsible editor：Li Xiang
Source：GMW.cn
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