Researchers from Temple University in Philadelphia, Pennsylvania, have announced that they have used a new gene-editing technique to remove the Human Immune Virus (HIV) from the genome of human immune cells. The scientists have published their findings in Nature Scientific Reports. Using the process known as CRISPR/Cas9 gene editing technique, the scientists were able to eliminate HIV-1 DNA from T cell genomes in laboratory experiments.
They also prevented re-infection after the cells were re-exposed to the virus. This means that they have effectively been able to shut down HIV replication permanently. It is a milestone achievement in finding a cure to HIV.
The gene editing technique works by guiding ‘scissor-like’ proteins to targeted sections of DNA within a cell, and then prompting them to alter or ‘edit’ them in some way. CRISPR refers to a specific repeating sequence of DNA extracted from a prokaryote – a single-celled organism such as bacteria – which pairs up with an RNA-guided enzyme called Cas9.
Science Alert reports that to edit the DNA of a virus within a human cell, a bacterium is needed to go in, encounter the virus, and produce a strand of RNA that is identical to the sequence of the virtual DNA. This will guide the RNA. It will then latch onto the Cas9 enzyme, and together, they will search for the matching virus. Once they locate it, the Cas9 cuts and destroy it.
By this technique, the researchers managed to eliminate HIV-1 DNA from T cell genomes in human lab cultures. The scientists said when these cells were later exposed to the virus, they were protected from re-infection.
Lead researcher of the study, Kamel Khalili said in a press statement that the study is a major breakthrough in an attempt to look for a cure to a disease that has caused much havoc around the world.
“The findings are important on multiple levels. They demonstrate the effectiveness of our gene editing system in eliminating HIV from the DNA of CD4 T-cells and, by introducing mutations into the viral genome, permanently inactivating its replication. Further, they show that the system can protect cells from re-infection and that the technology is safe for the cells, with no toxic effects. These experiments had not been performed previously to this extent. But the questions they address are critical, and the results allow us to move ahead with this technology,” Khalili wrote in the statement.
In the past, scientists have tried gene-editing techniques on HIV. However, this is said to be the first time they have been able to prevent further infections. Preventing further infection is said to be very crucial to the success of a treatment of HIV.
Preventing further infection is very effective than the use of antiretroviral drugs. This is because once patients stop taking the antiretroviral drugs, the HIV starts overloading the T-cells again.
Khalili said “Antiretroviral drugs are very good at controlling HIV infection. But patients on antiretroviral therapy who stop taking the drugs suffer a rapid rebound in HIV replication.”
According to Science Alert, CRISPR/Cas9 is what researchers in the United Kingdom have recently gotten approval to use on human embryos to be able to find how to improve In Vitro Fertilization (IVF) success rates and reduce miscarriages. IVF is the process of fertilization by manually combining an egg and sperm in a laboratory dish, and then transferring the embryo to the womb of a female.
In early 2016, scientists used CRISPR/Cas9 to successfully treat a genetic disease known as Duchenne muscular dystrophy in living mammals for the first time.
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