title: Targeted sequence alteration of a chromosomal locus in mouse liver.
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 approximately 0.1%. These results demonstrate the significant potential of this method for applications in gene therapy and biotechnology.
authors.affiliation: Faculty of Pharmaceutical Sciences, Hokkaido University, Kita-12, Nishi-6, Kita-ku, Sapporo 060-0812, Japan. hirokam@pharm.hokudai.ac.jp
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Kamiya
authors.forename: Masayuki
authors.initials: M
authors.lastname: Uchiyama
authors.forename: Jingshu
authors.initials: J
authors.lastname: Piao
authors.forename: Yoshimichi
authors.initials: Y
authors.lastname: Nakatsu
authors.forename: Teruhisa
authors.initials: T
authors.lastname: Tsuzuki
authors.forename: Hideyoshi
authors.initials: H
authors.lastname: Harashima
mesh_terms: D000818:Animals; D001483:Base Sequence; D001709:Biotechnology / Q000379:methods; D033481:Chromosomes, Mammalian / Q000235:genetics*; D004277:DNA, Single-Stranded / Q000008:administration & dosage*; D005260:Female; D017930:Genes, Reporter; D015316:Genetic Therapy / Q000379:methods; D016678:Genome; D008099:Liver / Q000378:metabolism*; D008297:Male; D051379:Mice; D008822:Mice, Transgenic; D017422:Sequence Analysis, DNA
chemical_list: D004277:DNA, Single-Stranded