Joseph E. Rabinowitz,James M. Wilson,Susan M. Faust
申请号：
US15099651
公开号：
US20160222414A1
申请日:
2016.04.15
申请国别(地区):
US
年份:
2016
代理人:
摘要：
A CpG-modified recombinant adeno-associated viral (AAV) vector is described. The vector carries a nucleic acid molecule comprising AAV inverted terminal repeat (ITR) sequences and an exogenous gene sequence under the control of regulatory sequences which control expression of the gene product, in which the nucleic acid sequences carried by the vector are modified to significantly reduce CpG di-nucleotides such that an immune response to the vector is reduced as compared to the unmodified AAV vector. Also provided are methods and regimens for delivering transgenes using these AAV viral vectors, in which the innate immune response to the vector and/or transgene is significantly modulated.
