单碱基编辑工具及在基因治疗中的应用及前景

Base Editing and its Applications in Gene Therapy

随着CRISPR/Cas9基因编辑工具效率的提高,其应用范围逐渐从实验室扩展到临床,但CRISPR/Cas9进行基因修改的效率还不够高。单碱基编辑工具直接将一种碱基对永久转变成另一种碱基对,是一种不需要切开双链DNA的化学修复方法,可以高效地进行基因修复。尽管单碱基编辑工具研发时间不长,但是已经在基因编辑领域得到了广泛的应用,包括在小鼠内耳的基因编辑。单碱基编辑工具将是开展耳聋基因治疗的有效工具。

The improvement of CRISPR/Cas9 gene editing efficiency has promoted its applications in bench and clinic research.However,the efficiency of CRISPR/Cas9 is not sufficient for gene modification.Base editing can convert one base pair to another permanently and efficiently,without double strains break.Although base editing was only recently developed,it has gained widespread utilization in gene editing,including in mouse inner ear models.Base editing is an effective tool for inner ear gene therapy.