TY - JOUR
T1 - Targeted sequence alteration of a chromosomal locus in mouse liver
AU - Kamiya, Hiroyuki
AU - Uchiyama, Masayuki
AU - Piao, Jingshu
AU - Nakatsu, Yoshimichi
AU - Tsuzuki, Teruhisa
AU - Harashima, Hideyoshi
N1 - Funding Information:
This work was supported in part by Grants-in-Aid from the Ministry of Education, Culture, Sports, Science and Technology of Japan and from the Japan Society for the Promotion of Science.
PY - 2010/3
Y1 - 2010/3
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=77649187509&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=77649187509&partnerID=8YFLogxK
U2 - 10.1016/j.ijpharm.2009.12.020
DO - 10.1016/j.ijpharm.2009.12.020
M3 - Article
C2 - 20025952
AN - SCOPUS:77649187509
SN - 0378-5173
VL - 387
SP - 180
EP - 183
JO - International Journal of Pharmaceutics
JF - International Journal of Pharmaceutics
IS - 1-2
ER -