Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase

使用实验室进化的 CRISPR 相关转座酶在人类细胞中进行可编程基因插入

关键词：

来源：

science

来源地址：

https://www.science.org/doi/10.1126/science.adt5199

资源所属：

马铃薯基因组设计育种

类型：

前沿资讯

语种：

英语

原文发布日期：

2025-05-15

摘要：

The ability to install large DNA sequences into specified locations in the human genome has far-reaching implications, including paving the way for single-drug treatments of mutationally diverse loss-of-function genetic diseases. CRISPR-associated transposases (CASTs) are naturally occurring systems that support RNA-programmable insertion of gene-sized DNA but have shown minimal activity in human cells. Witte et al. developed a continuous evolution platform to improve CAST activity, yielding an evolved CAST with more than 200-fold increased activity in human cells. This enzyme enables efficient gene integration across a variety of therapeutically relevant genomic sites in multiple human cell types, representing a versatile new platform for mammalian cell genome editing.