A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo

摘要：

Dear Editor,The clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) system has revolutionized biomedical research and facilitated the development of new therapies based on genome editing ”1”.A major roadblock to achieve the therapeutic potential of the CRISPR/Cas system is the lack of a safe and effectiving in vivo delivery method.Adeno-associated virus (AAV)-assisted delivery of the CRISPR/Cas9 system has shown gene targeting efficacy in vivo,however,the long persistence and immunogenicity of AAV in the host prevent the wide therapeutic application of AAV-based CRISPR/Cas9 delivery ”2”.

机标关键词：therapeutic potential、biomedical research、long persistence、gene targeting

所属期刊栏目：27

分类号：R6 ;R3

资助基金：grants from the State Key Research Development Program of China2016YFC1200300;the Young Thousand Talent Program and the Bill & Mellinda Gates Foundation to XT.YD acknowledges the following supports:the Early Career Investigator Award from the Bayer Hemophilia Awards Program,Research Awards from the National PKU Alliance,New Investigator Grant from the AAPS Foundation,Maximizi

在线出版日期：2017-04-14（万方平台首次上网日期，不代表论文的发表时间）

页数：共4页

页码：440-443