Targeted sequence alteration of a chromosomal locus in mouse liver

Hiroyuki Kamiya, Masayuki Uchiyama, Jingshu Piao, Yoshimichi Nakatsu, Teruhisa Tsuzuki, Hideyoshi Harashima

Research output: Contribution to journalArticlepeer-review

10 Citations (Scopus)


Targeted 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.

Original languageEnglish
Pages (from-to)180-183
Number of pages4
JournalInternational Journal of Pharmaceutics
Issue number1-2
Publication statusPublished - Mar 2010

All Science Journal Classification (ASJC) codes

  • Pharmaceutical Science


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