TEMPLE UNIVERSITY - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
发明人:
KHALILI, KAMEL,HU, WENHUI,KAMINSKI, RAFAL,MALCOLM, THOMAS
申请号:
CA3026332
公开号:
CA3026332A1
申请日:
2017.05.26
申请国别(地区):
CA
年份:
2017
代理人:
摘要:
A CRISPR-endonuclease gene editing composition includes a guide RNA (gRNA) for targeting a specific viral sequence for cleavage by the endonuclease which introduces breaks in the double stranded DNA identified by the gRNA. Placing the gene encoding Cas9 under the control of a minimal promoter of, for example, HIV spanning the 5'-LTR, results in the activation by the HIV-1 transactivator protein, Tat. Co-expression of both a multiplex of, for example, HIV-specific gRNAs and endonuclease, e.g. Cas9, in cells results in the modification and/or excision of the segment of viral DNA, leading to the eradication of the virus in vitro and in vivo.