Targeted sequence alteration of a chromosomal locus in mouse liver

• Kamiya, Hiroyuki

• Uchiyama, Masayuki

• Piao, Jingshu

• Nakatsu, Yoshimichi

• Tsuzuki, Teruhisa

• Harashima, Hideyoshi

抄録

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.

収録刊行物

• International Journal of Pharmaceutics

  International Journal of Pharmaceutics 387 (1-2), 180-183, 2010-03-15

  Elsevier B.V.

キーワード

• Targeted sequence alteration
• Gene correction
• Tailed duplex DNA fragment
• rpsL transgenic mouse
• Hydrodynamic tail vein injection

詳細情報

• CRID: 1050282813982500608
• NII論文ID: 120002015024
• HANDLE: 2115/42890
• ISSN: 03785173
• 本文言語コード: en
• 資料種別: journal article
• データソース種別:
  • IRDB
  • CiNii Articles