CRISPR-Cas9

CRISPR, short for CRISPR-Cas9, is a technology that has the potential to change the world in the very near future. CRISPR is starting to become of great interest not only to scientists and researchers but to many people fascinated by the vast number of benefits it has to offer human health. Some of the health benefits include an efficient anti-aging treatment, the elimination of genetic disorders, and being a potential cure for life threatening cancers and horrific autoimmune disorders such as Multiple Sclerosis.

So, what is CRISPR? In the simplest terms CRISPR is used to edit human genomes, thus allowing scientists and researchers to alter DNA sequences and modify gene function easily and efficiently. CRISPR in its original form is basically a homing device the that guides the Cas9 enzyme as if they were molecular scissors that target specific sections of DNA. The CRISPR and Cas9 will then work together as a genetic-engineering cruise missile that deactivates, replaces, or repairs a gene. It can also insert something completely new where the Cas9 scissors cut something out.

Currently this technology is being studied for and can be used for the treatment of cancer, stopping the spread of disease such as the flu, and correcting genetic defects. The thing that makes CRISPR have so much potential in opposition to other technologies is its simplicity. It effortlessly finds a particular part of DNA inside of a cell, then it alters that piece of DNA. Not only can CRISPR alter DNA it can also turn genes on or off without altering their sequence at all. This means that scientists can go in and target a mutation that causes a disorder and simply delete it or repair it. An example of this would be sickle cell anemia. This disorder is caused by one mutation in a gene out of the approximately 20,000 we have in our DNA. Sickle cell anemia is an extremely painful disorder, that changes a person’s red blood cells to a sickle shape, causing them to get stuck in clumps in the vessels and cause extreme pain. Currently the disorder is treated by monthly blood infusions that can only offer temporary relief and have potentially deadly side effects. So, what if instead of giving the patient a treatment that is ongoing, uncomfortable, and painful we could offer a one-time procedure that goes in and completely erases the problem? This is what many researchers and scientists believe they can do with this CRISPR. This would save people around the world millions of dollars in hospital bills and allow people who are suffering from horrible disorders to live a normal and pain free life.

Research done by the Saulk Institute in 2019 shows promise to it also being an effective anti-aging treatment. The article that was published on February 18, 2019 in the journal Nature Medicine, shows a novel CRISPR-Cas9 genome-editing therapy that was able to stifle the accelerated aging process in mice that had Hutchinson-Gilford Progeria Syndrome. This rare genetic disorder also affects and is deadly to humans. The disorder causes a rapid acceleration of the aging process and is ultimately life threatening. CRISPR-Cas9 had successful results with editing the mutation causing this disorder on mice resulting in a deceleration of aging. It is important to note that Hutchinson-Gilford Progeria Syndrome creates an accelerated aging process that is abnormal. They did not yet efficiently reverse or halt typical aging of a healthy adult. Regardless, this treatment provides important insight into the molecular pathways involved in normal and accelerated aging. It also gives us a look into ways to reduce toxic proteins through the use of gene therapy.

This information leads us to many exciting questions. Could we potentially figure out a way to halt or reverse aging and eliminate disease that comes with getting older? Could we someday see our loved ones walk again that were burdened with MS and Parkinson’s? Could we regain the energy that we once had in our twenties and avoid life threatening disease such as cancer? It really seems like the sky is the limit with CRISPR and that we may see some of these advancements in the very near future.