'Prime Editing' to Cure Genetic Diseases... Accuracy Verification

Potential as a Gene Therapy Agent

[Asia Economy Reporter Hwang Junho] A domestic research team has verified the efficiency and accuracy of the next-generation gene-editing technology called 'Prime Editing.' It is expected that the gene therapy technology using Prime Editing, which is anticipated to treat up to 90% of genetic diseases, will have increased potential for application.

The research team led by Dr. Kim Daesik and Dr. Kim Yongsam from the Gene Editing Research Center at the Korea Research Institute of Bioscience and Biotechnology announced on the 10th that they published a research paper verifying the efficiency and accuracy of Prime Editing in the international academic journal 'Nucleic Acids Research.'

Verification of the Efficiency and Accuracy of Prime Editing

Prime Editing is a gene-editing technology first announced in October last year by the Broad Institute in the United States. It is a method that uses the existing third-generation gene-editing technology CRISPR/Cas9 and reverse transcriptase enzyme to perform gene correction. The conventional method works by cutting both strands of DNA and allowing the cell to naturally repair it, whereas Prime Editing cuts a single strand of DNA and directly inserts the desired nucleotide sequence. The academic community expects that this technology could potentially treat up to 90% of rare genetic diseases.

The research team developed a technique to verify the accuracy of Prime Editing. They examined various possibilities of off-target cuts that could occur in Prime Editing by utilizing mismatches in the extended 'guide RNA (pegRNA)' used in Prime Editing. They also demonstrated the importance of single-strand DNA cutting and the efficiency and accuracy depending on the length of the extended guide RNA. Furthermore, they measured the efficiency and accuracy of Prime Editing variants created using Cas9 mutants and confirmed that these variants have higher accuracy compared to conventional Prime Editing.

Prime Editing Increases Potential for Treating Rare Genetic Diseases

This study is the world's first to verify the efficiency and accuracy of Prime Editing. Dr. Kim Daesik, the principal investigator, stated, "By verifying the high efficiency and accuracy of Prime Editing, we have demonstrated its potential as a gene therapy agent," adding, "By verifying the efficiency and accuracy of Prime Editing, which has a wide range of applications, various gene corrections are possible with a single gene-editing technology, and together with the development of auxiliary technologies such as delivery methods, the potential for gene therapy utilization has increased."

This content was produced with the assistance of AI translation services.