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학생구두발표

A Universal Gene Editing Technology (UGET) : Genome engineering using a nucleotide-probed, dimerized FokI endonuclease module

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.

저자정보

  • Seong Kook Jeon Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Jin Suk Park Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Jeong Gu Kang Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Sun Hee Kim Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Nan Ee Lee Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Jung Mi Lee Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Subin Moon Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Dae In Ha Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Do Yon Kim Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Jeong-Heon Ko Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea
  • Yong-Sam Kim Genome Editing Research Center, KRIBB, 125 Gwahak-ro, Daejeon 34141, South Korea

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