Targeted sequence alteration of a chromosomal locus in mouse liver
Targeted sequence alteration of a chromosomal locus in mouse liver
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DOI：
10.1016/j.ijpharm.2009.12.020
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发表时间：
2010-03-15
影响因子：
5.8
通讯作者：
Harashima, Hideyoshi
中科院分区：
文献类型：
--
作者：
Kamiya, Hiroyuki;Uchiyama, Masayuki;Harashima, Hideyoshi
靶向序列改变将成为基因治疗和生物技术的一种有吸引力的方法。为了实现体内靶向序列的改变，用尾静脉流体动力法将35mer和794mer单链DNA组成的拖尾双链DNA注射到基因组中含有报告基因(rpsL基因)的转基因小鼠的肝脏中。在rpsL转基因的80位密码子上，拖尾DNA被设计用于ATC到AGC的转换。在肝脏转基因中诱导了预期的T->；G序列改变，其效率类似于0.1%。这些结果表明，这种方法在基因治疗和生物技术中具有巨大的应用潜力。(C)2010爱思唯尔B.V.保留所有权利。
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 similar to 0.1%. These results demonstrate the significant potential of this method for applications in gene therapy and biotechnology. (C) 2010 Elsevier B.V. All rights reserved.