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Targeted sequence alteration of a chromosomal locus in mouse liver

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Title: Targeted sequence alteration of a chromosomal locus in mouse liver
Authors: Kamiya, Hiroyuki Browse this author
Uchiyama, Masayuki Browse this author
Piao, Jingshu Browse this author
Nakatsu, Yoshimichi Browse this author
Tsuzuki, Teruhisa Browse this author
Harashima, Hideyoshi Browse this author →KAKEN DB
Keywords: Targeted sequence alteration
Gene correction
Tailed duplex DNA fragment
rpsL transgenic mouse
Hydrodynamic tail vein injection
Issue Date: 15-Mar-2010
Publisher: Elsevier B.V.
Journal Title: International Journal of Pharmaceutics
Volume: 387
Issue: 1-2
Start Page: 180
End Page: 183
Publisher DOI: 10.1016/j.ijpharm.2009.12.020
PMID: 20025952
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.
Type: article (author version)
Appears in Collections:薬学研究院 (Faculty of Pharmaceutical Sciences) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)

Submitter: 紙谷 浩之

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