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 journalArticle

5 Citations (Scopus)

Abstract

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
Volume387
Issue number1-2
DOIs
Publication statusPublished - Mar 1 2010

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Biotechnology
Transgenes
Genetic Therapy
Single-Stranded DNA
Liver
DNA
Hydrodynamics
Reporter Genes
Codon
Transgenic Mice
Tail
Veins
Genome
Injections
Genes

All Science Journal Classification (ASJC) codes

  • Pharmaceutical Science

Cite this

Targeted sequence alteration of a chromosomal locus in mouse liver. / Kamiya, Hiroyuki; Uchiyama, Masayuki; Piao, Jingshu; Nakatsu, Yoshimichi; Tsuzuki, Teruhisa; Harashima, Hideyoshi.

In: International Journal of Pharmaceutics, Vol. 387, No. 1-2, 01.03.2010, p. 180-183.

Research output: Contribution to journalArticle

Kamiya, Hiroyuki ; Uchiyama, Masayuki ; Piao, Jingshu ; Nakatsu, Yoshimichi ; Tsuzuki, Teruhisa ; Harashima, Hideyoshi. / Targeted sequence alteration of a chromosomal locus in mouse liver. In: International Journal of Pharmaceutics. 2010 ; Vol. 387, No. 1-2. pp. 180-183.
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