Constructing and packaging scAAV vectors and detecting their transduction efficiency  

作　　者：Jianqing Wu Weihong Zhao 

机构地区：[1]Department of Geriatrics [2]Department of Nephrology the First Affiliated Hospital of Nanjing Medical University Nanjing 210029, Jiangsu Province, China

出　　处：《Journal of Nanjing Medical University》2007年第6期401-401,共1页南京医科大学学报（英文版）

基　　金：This study was supported by the Natural Science Foundation of Jiangsu Province(BK2006244);the Key Medical Person Foundation of Jiangsu Province (RC2007051)

摘　　要：Gene therapy is a form of molecular medicine that promises to provide new treatments for a large number of inherited and acquired diseases. One of the biggest stumbling blocks to successful widespread application of genetic treatments is the development of safe and effective vectors with which to ferry genetic material into cells. Adeno-associated virus （AAV）, a nonpathogenic human parvovirus, has gained attention as a potentially safe vector for gene transfer and gene therapy. The AAV genome is a single-stranded DNA, which is transcriptionally inactive. It has been reported that viral second-strand DNA synthesis is the rate-limiting step in AAV-mediated transgene expression. Self-complementary adeno-associated viral（scAAV） vectors bypass the requirement for viral second-strand DNA synthesis, and lead to a significant increase in transduction efficiency, scAAV vectors have been successfully constructed by Xiao and Smulski in the USA in 2003. Recently, we have developed and packaged scAAV, and reported as following.Gene therapy is a form of molecular medicine that promises to provide new treatments for a large number of inherited and acquired diseases. One of the biggest stumbling blocks to successful widespread application of genetic treatments is the development of safe and effective vectors with which to ferry genetic material into cells. Adeno-associated virus （AAV）, a nonpathogenic human parvovirus, has gained attention as a potentially safe vector for gene transfer and gene therapy. The AAV genome is a single-stranded DNA, which is transcriptionally inactive. It has been reported that viral second-strand DNA synthesis is the rate-limiting step in AAV-mediated transgene expression. Self-complementary adeno-associated viral（scAAV） vectors bypass the requirement for viral second-strand DNA synthesis, and lead to a significant increase in transduction efficiency, scAAV vectors have been successfully constructed by Xiao and Smulski in the USA in 2003. Recently, we have developed and packaged scAAV, and reported as following.

关 键 词：gene therapy self-complementary adeno-associated viral transduction efficiency 

分 类 号：R1[医药卫生—公共卫生与预防医学]