CRISPR/Cas9-Based Gene Editing in HIV-1/AIDS Therapy

elzway, tamiem (2020-03-13)

The human immunodeficiency virus (HIV) is the cause of acquired immunodeficiency syndrome (AIDS) belongs to the lentivirus subgroup of retroviruses which formed by single-strand RNA molecules. The complete genome is flanked by two long terminal repeat (LTR) sequences and it encodes 10 viral proteins including gag, pol, vif, vpr, vpu, env, tat, rev, nef, and the antisense protein (ASP), which have different functions in virus invasion and replication (1) . There are two different types of HIV, HIV-1, and HIV-2. They both have many similarities and both can lead to AIDS. Compared to HIV-1, HIV-2 has lower transmissibility and is less pathogenic. HIV-1 is recognized as the major cause of AIDS and becomes the main target to prevent and cure AIDS


HIV is one of the main infectious diseases that infect millions of people around the world and the major cause of AIDS, even though great efforts have been made in the prevention and therapy of HIV-1 infection, HIV-1/AIDS remains a major threat to global human health. Highly active antiretroviral therapy (HAART) can suppress virus replication, but it cannot eradicate latent viral, making the HIV-1/AIDS a chronic and persistence of the virus in latent reservoirs, which includes macrophages, microglia, astrocytes, intestinal lymphoid cells, and, mainly, CD4+ memory lymphocytes and failure of patient adhesion to treatment. More Recently scientist found powerful genetic tool called the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated nuclease 9 (Cas9) system has been engineered as a more effective gene-editing and cheaper technology with the potential to treat HIV-1/AIDS by targeting the long terminal repeats and the Gag gene for elimination of latent virus infection without off-target effect, which are excised in Vivo using infected mice. The integrated Pro-viral DNA elimination confirmed by digital droplet PCR with no off-target effect is detected. These data provide proof of concept that permanent viral elimination is possible without viral rebound.

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