The invention discloses an engineered
exosome targeting
delivery system (EMT-Cas12a) and application thereof in AIDS (acquired
immune deficiency syndrome) treatment. The targeting
delivery system is loaded with a functional component for
cutting HIV (
human immunodeficiency virus) previrus
DNA (deoxyribonucleic acid) on the basis of
CRISPR-Cas12a (clustered regularly interspaced short palindromic repeats). According to the targeting
delivery system, a nano
antibody Nb1 of targeting CD4 is expressed on the surface of EMT-Cas12a, so that the efficiency of targeting delivery of
CRISPR-Cas12a to CD4 + cells is improved. According to the targeted delivery
system, a multi-crRNA series strategy is designed, a conserved region shared by a targeted HIV-1 B subtype and Chinese epidemic strains (07BC, 08BC and B ') is conserved, the crRNA and Cas12a mRNA form a functional component for
cutting HIV previrus
DNA, the functional component and Cas12a mRNA are jointly packaged into EMT-Cas12a, and the risk of
virus escape is reduced. According to the targeting delivery
system, in cells cultured
in vitro (
cell lines infected with HIV-1 and PBMC derived from HIV-1 positive donors), and
in vivo tests of intravenous injection of EMT-Cas12a in humanized mice infected with HIV-1, good
biological safety characteristics and excellent antiviral
efficacy are proved.