Gene Therapy for HIV/AIDS – IP 1659

HIV infection in human cells is either active or a latent.In most human cells, the HIV infection is active; however, in rare human cells,latent infection can occur at very early stage. These very small numbers oflatently infected cells called HIV reservoirs are located mainly in brain,peripheral blood, and lymphoid tissue. In the reservoir cell sites antiviraldrugs penetration is often low. Moreover, even under antiretroviral therapy(ART), about 30 to 50% of AIDS patients eventually develop HIV-associatedneurological disorders (HAND), which are cognitive, motor and/or behavioralimpairments caused by HIV infection in human brain. The ultimate cure forHIV/AIDS would be the removal or disruption of integrated HIV provirus inlatently infected cells or the complete elimination of these latent cells.However, gene therapy for HIV/AIDS has progressed very slowly. FIU inventors have developed compositioncomprising a CRISPR-Cas protein or a CRISPR-Cas plasmid encoding a Cas9protein, and one or more gRNAs that are effective for removing or disrupting anHIV provirus genome integrated into the genome of a cell. These compositionscan be encapsulated in carriers, for example, liposomes, wherein the liposomescarry on their surface one or more binding agents that bind specificallymolecules present on the surface of the target cells. The binding agentspresent on the surface of liposomes can be an aptamer, an antibody or anantigen binding fragment thereof, a ligand, etc., magnetic nanoparticles, andliposomes. The compositions can also comprise magnetic nanoparticles for braintargeting and blood brain barrier transmigration. Anne Laure Schmitt Olivier 305-348-5948

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