2024-03-28T22:14:59Zhttps://eprints.lib.hokudai.ac.jp/dspace-oai/requestoai:eprints.lib.hokudai.ac.jp:2115/428902022-11-17T02:08:08Zhdl_2115_20044hdl_2115_124Targeted sequence alteration of a chromosomal locus in mouse liverKamiya, HiroyukiUchiyama, MasayukiPiao, JingshuNakatsu, YoshimichiTsuzuki, TeruhisaHarashima, HideyoshiTargeted sequence alterationGene correctionTailed duplex DNA fragmentrpsL transgenic mouseHydrodynamic tail vein injection499Targeted sequence alteration would be an attractive method in gene therapy and biotechnology. To achieve in vivo targeted sequence alteration, a tailed duplex DNA consisting of annealed 35mer and 794mer single-stranded DNAs was delivered by means of hydrodynamic tail vein injection into liver of transgenic mouse harboring a reporter gene (the rpsL gene) in its genome. The tailed DNA was designed for a conversion of ATC to AGC at codon 80 of the rpsL transgene. The anticipated T → G sequence alteration was induced in the transgene in the liver with an efficiency of ∼0.1%. These results demonstrate the significant potential of this method for applications in gene therapy and biotechnology.Elsevier B.V.Journal Articleapplication/pdfhttp://hdl.handle.net/2115/42890https://eprints.lib.hokudai.ac.jp/dspace/bitstream/2115/42890/1/IJP387-1-2_180-183.pdf0378-5173International Journal of Pharmaceutics3871-21801832010-03-15enginfo:pmid/20025952info:doi/10.1016/j.ijpharm.2009.12.020author