Gene Therapy for HIV/AIDS


Florida International University (FIU) is seeking a business partner to develop and commercialize composition comprising a CRISPR-Cas protein and one or more gRNAs for treating, suppressing, and eradicating latent human immunodeficiency virus (HIV) infection.

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 of latently infected cells called HIV reservoirs are located mainly in brain, peripheral blood, and lymphoid tissue. In the reservoir cell sites antiviral drugs penetration is often low. Moreover, even under antiretroviral therapy (ART), about 30 to 50% of AIDS patients eventually develop HIV-associated neurological disorders (HAND), which are cognitive, motor and/or behavioral impairments caused by HIV infection in human brain. The ultimate cure for HIV/AIDS would be the removal or disruption of integrated HIV provirus in latently infected cells or the complete elimination of these latent cells. However, gene therapy for HIV/AIDS has progressed very slowly.

FIU inventors have developed composition comprising a CRISPR-Cas protein or a CRISPR-Cas plasmid encoding a Cas9 protein, and one or more gRNAs that are effective for removing or disrupting an HIV provirus genome integrated into the genome of a cell. These compositions can be encapsulated in carriers, for example, liposomes, wherein the liposomes carry on their surface one or more binding agents that bind specifically molecules present on the surface of the target cells. The binding agents present on the surface of liposomes can be an aptamer, an antibody or an antigen binding fragment thereof, a ligand, etc., magnetic nanoparticles, and liposomes. The compositions can also comprise magnetic nanoparticles for brain targeting and blood brain barrier transmigration.

  • Treatment, suppression, and eradication of latent HIV infection
  • Can be specifically delivered to target cells without affecting the non-targeted cells
  • Represents a new advanced therapeutic strategy to eliminate HIV provirus from HIV latent cells

Call Anne-Laure “Anlo” Schmitt-Olivier at 305-348-5948 or fill out the quick contact form below.