원문정보
초록
영어
Genome editing technology promises to provide versatile tools for the generation of various model cell lines, plants and animals as well as for gene therapy by gene-editing in a target-specific manner. To date, three distinct modes of genome editing technologies have been introduced and extensively investigated in experimental settings, and attempted for use in clinical settings. Despite the revolutionized efficiency and sophistication in gene editing owing to development of CRISPR/Cas9, there remains technical limitations for currently available programmable nucleases in the routine use in experimental and clinical settings. Here, we introduce a universal genome editing technology (UGET) that relies on gene targeting through simple base pairings between a 20-28 nt nucleotide probe and a stretch of single-strand target DNA at the replication fork. UGET has no target limitations, shows lower-than-expected off-target incidence, is straightforward to use, and is compatible to gene correction via HDR. In particular, an overall construct size is less than 2.3kb, which means a high flexibility for use in gene therapy using viral vectors including adenovirus-associated viruses as well as for agrobacterium-based transformation in plants.