Gene Editing Therapy – A Step Closer Cure for HIV/AIDS
The main objective of the gene editing therapy in finding a cure for HIV/AIDS is contributing a set of new immune cells that will resist HIV-infection and reconstitute the immune system.
The use of antiretroviral therapy (ART) has partially succeeded in controlling virus infection in most infected individuals. ART enhances health and elongates the lives of individuals infected with HIV.
In addition, it also lowers the transmission rates of the virus from one individual to another. However, this treatment regimen is linked with several comorbidities, has very little impact on complete elimination of HIV reservoir and needs patients to comply with a lifelong drug routine.
Even though HIV-infected individuals using ART will have very small concentrations of the virus in their plasma, the latently infected CD4+ T cells still exist.
The use of antiretroviral therapy (ART) prevents the multiplication of the HIV virus but cannot completely cure it.
The virus tends to rebound when the therapy is stopped because of the viral reservoir which persists in the body. This viral reservoir is the major barrier to finding a cure for HIV infection.
However, there is a small number of people who are resistant to HIV due to a natural mutation in their CCR5 gene.
This mutation, referred to as the CCR5-delta32, inhibits the HIV virus from accessing the body cells and causing infection.
Most strategies today are focused specifically on the virus, for example,
- Using the CRISPR technology which aims at the integrated HIV genome.
- Gene editing therapy is an exciting technology which opens up a wide range of pathways that scientists can explore. Scientists can identify novel genes that are needed for the virus to reproduce and determine the genetic mutations that make some people resistant to HIV infection.
The gene editing therapy can help in eradicating the HIV DNA from infected patients.
However, it is highly potent and flexible and can be used with the ARV therapy to promote further suppression of the viral RNA.
It can also be modified to target any mutated strains of the HIV virus.
Research carried out by Dr. Khalili from Temple University has established that the gene editing system that is based on CRISPR technology can eliminate the HIV Virus from human infected cells in vitro, without affecting the host cells.
Additionally, ev vivo experiments confirm that viral replication is significantly lowered after treatment with gene editing therapies.
Results from this study showed that portion of targeted HIV DNA was cut out from the HIV genome in all tissues.
This includes cells in the heart, brain, spleen, kidney, lungs, liver and plasma. There was also a significant reduction in the concentration of the HIV RNA in the lymphocytes and lymph nodes.
The ability of this rAAV delivery system to access many organs that contain the HIV genome and edit the viral DNA is a crucial indication that this therapy can overcome the reactivation of the HIV virus from the latently infected cells.
In addition, it can serve as a potentially curative approach for HIV-infected patients.
The gene editing technology has opened up countless possibilities for many researchers globally.
Apart from HIV/AIDS, this technology can be used to find treatment for other infections like autoimmune diseases, cancer, and infectious diseases.
This therapy can potentially help indirectly studying various phenomena in the human cells and will give scientists a better approach for curing different ailments affecting human beings.
Andrew Thompson is a scientific researcher who has been working on various research projects for HIV cure. Andrew has more than 30 years of research experience. For more information on HIV gene editing therapies, check out his website here.