Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.
Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.
复制标题
腺病毒载体用于体内CRISPR-CAS基因编辑器的体内传递。
DOI：
10.1016/j.jconrel.2020.09.003
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发表时间：
2020-11-10
期刊：
影响因子：
--
通讯作者：
Curiel DT
中科院分区：
文献类型：
--
作者：
Boucher P;Cui X;Curiel DT
Harnessing the bacterial clustered regularly interspaced short palindromic repeats (CRISPR) system for genome editing in eukaryotes has revolutionized basic biomedical research and the translational sciences. The ability to create targeted alterations of the genome through this easy to design system has presented unprecedented opportunities to treat inherited disorders and other diseases such as cancer through gene therapy. A major hurdle is the lack of an efficient and safe in vivo delivery system, limiting most of the current gene therapy efforts to ex vivo editing of extracted cells. Here we discuss the unique features of adenoviral vectors that enable tissue specific and efficient delivery of the CRISPR-Cas machinery for in vivo genome editing.
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