Abstract：
Base editor (BE) is a gene-editing tool developed by combining the CRISPR/Cas system with an individual deaminase, enabling precise single-base substitution in DNA or RNA without generating a DNA double-strand break (DSB) or requiring donor DNA templates in living cells. Base editors offer more precise and secure genome-editing effects than other conventional artificial nuclease systems, such as CRISPR/Cas9, as the DSB induced by Cas9 will cause severe damage to the genome. Thus, base editors have important applications in the field of biomedicine, including gene function investigation, directed protein evolution, genetic lineage tracing, disease modeling, and gene therapy. Since the development of the two main base editors, cytosine base editors (CBEs) and adenine base editors (ABEs), scientists have developed more than 100 optimized base editors with improved editing efficiency, precision, specificity, targeting scope, and capacity to be delivered in vivo, greatly enhancing their application potential in biomedicine. Here, we review the recent development of base editors, summarize their applications in the biomedical field, and discuss future perspectives and challenges for therapeutic applications.
摘要：
BaseEditor（BE）是通过将CRISPR/Cas系统与单独的脱氨酶相结合而开发的基因编辑工具，在DNA或RNA中实现精确的单碱基取代，而不产生DNA双链断裂（DSB）或需要活细胞中的供体DNA模板。Base编辑器提供比其他传统人工核酸酶系统更精确和安全的基因组编辑效果。如CRISPR/Cas9，因为Cas9诱导的DSB会对基因组造成严重的损伤。因此，基础编辑在生物医学领域有着重要的应用,包括基因功能调查，定向蛋白质进化，遗传谱系追踪，疾病建模,和基因治疗。自从两个主要的基础编辑器开发以来，胞嘧啶碱基编辑器（CBE）和腺嘌呤碱基编辑器（ABE），科学家开发了100多个优化的基础编辑器，提高了编辑效率，精度,特异性，瞄准范围，以及在体内传递的能力，大大提高了其在生物医学领域的应用潜力。这里,我们回顾了基础编辑的最新发展，总结它们在生物医学领域的应用，并讨论治疗应用的未来前景和挑战。
