Abstract
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.
Benefit
Can be specifically delivered to target cells without affecting the non-targeted cellsRepresents a new advanced therapeutic strategy to eliminate HIV provirus from HIV latent cells
Market Application
Treatment, suppression, and eradication of latent HIV infection
Brochure
