Lab mouse

As biology makes the transition to an information technology we are starting to see a marked acceleration in its progress. Just two years ago CRISPR was essentially unheard of outside of academia, today it’s underpinning a revolution in genetic engineering that has the potential to treat a myriad of diseases including cancer and Alzheimer’s. Thousands of labs around the world are now employing CRISPR daily in their research.

Now CRISPR may be on the path of making antiretroviral drugs a thing of past for treating patients suffering from HIV. Scientists from the US and China working in collaboration have published a paper in the journal Molecular Therapy demonstrating the removal of the HIV-1 virus from infected cells in mice and the complete halting of its further replication using their engineered CRISPR/Cas9 tool. Their data shows that they were able to overcome one of the biggest hurdles in treating HIV which has been the virus’ capacity to hide inside a cell’s DNA and most importantly inside the DNA of the T-cells that should be combating it. The authors report that only a single application of their engineered CRISPR/Cas9 was required in order to remove the HIV-1 DNA from the infected cells.

Even more impressive is the fact that this research was done with genetically engineered “humanized” mice that contained human T-cells. Although this study was only testing the efficacy of the technique there was some preliminary evidence of its safety as no off-target cuts were detected in the cells of the treated mice. Off-target cuts have the potential to induce dangerous mutations that could potential cause cancer and have plagued CRISPR and other forms of genetic engineering in the past.

The researchers say their next goal is to demonstrate similar efficacy of HIV-1 removal in a study using primate models. In the primate study they will look to see if they can demonstrate the removal of the HIV-1 DNA from the brain which is currently a key question. The primate study will also look at the safety of the technique. If the primate study proves successful the researchers will they begin the first stages of testing their technique in human patients with HIV. This research could also act as a proof-of-principle for treating genetic disorders such as cystic fibrosis, sickle cell anemia, hemophilia etc.

George Church professor of Genetics at Harvard and a key CRISPR researcher says that while as amazing as CRISPR is he predicts that even more accurate and easier to use gene editing tools are possible in the near future. Professor Church points to emerging gene editing tools such as the protein NgAgo that researchers claim (although there is some academic controversy) can accurately edit genomes in mammals without the need for a guide RNA.

At the current pace of progress it would be very surprising if gene editing via CRISPR or competing techniques does not become a routine method for treating a wide range of pathologies by the mid 2020s.


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