Creating zinc finger nucleases to manipulate the genome in a site-specific manner using a modular-assembly approach.
Creating zinc finger nucleases to manipulate the genome in a site-specific manner using a modular-assembly approach.
复制标题
使用模块化组装方法创建锌指核酸酶以位点特异性方式操纵基因组。
DOI:
10.1101/pdb.top93
复制
发表时间:
2010
影响因子:
--
通讯作者:
Porteus,Matthew
中科院分区:
文献类型:
--
作者:
Porteus,Matthew
BACKGROUNDIn this article, gene targeting is defined as the transfer of genetic information from an introduced fragment of DNA to the genome by homologous recombination. It represents the most precise way to manipulate the genome and can be used to introduce changes both small (eg, single nucleotide changes) and large (such as the introduction of several thousand base pairs of new DNA) into the genetic material of a cell. Gene targeting has been a powerful experimental tool in the study of bacteria, yeast, murine embryonic stem cells (Capecchi 1989), and certain other specialized vertebrate cell lines such as the chicken DT40 cell line (Buerstedde and Takeda 1991). Moreover, because of its precision, gene targeting would be an ideal way to perform gene therapy. However, the broad experimental use of gene targeting in vertebrate cells and its use for therapeutic purposes have been precluded by its low spontaneous rate. In HEK-293 cells, for example, the spontaneous rate of gene targeting is approximately one event per million cells transfected (Porteus and Baltimore 2003).