Prime editing and its application in the treatment of diseases

Abstract. With the rapid development of biotechnology, gene editing technologies have become an important research direction in the field of biomedicine, and their potential application in disease treatment has become increasingly prominent. As a new type of gene editing technology, prime editors have received widespread attention. In recent years, gene editing technologies have made remarkable progress. For example, CRISPR-Cas9 system has been widely used, which provides new ideas and methods for the treatment of hereditary diseases, cancers, etc. The application scope of PE is even wider, which enables researchers to successfully achieve the intervention and treatment of many diseases by precisely editing the target genes. Although the application of PE in disease treatment shows great potential, there are still technical challenges in its editing efficiency as well as delivery efficiency, which need to be further studied and explored. The text in this article analyses the composition, principles, and optimization strategies of PE, followed by an in-depth discussion of its application in the treatment of Hereditary tyrosinaemia type (HT1) and 1-antitrypsin deficiency(A1ATD). By reviewing and analysing the use of PE in different diseases, this paper reveals the potential and challenges of PE and looks into its future. The study in this paper provides a reference for the use of PE in editing efficiency enhancement as well as in disease treatment. However, there are still many issues that need to be addressed, such as improving the accuracy and efficiency of PE and reducing off-target effects. Future research can focus on these directions to further promote the application and development of gene editing technologies in the medical field.