By Wajiha Paracha
How advancing gene therapy can help us change our luck.
Gene are what makes us who we are, by that I refer to both internal and external features. They determine our capabilities and how well we can perform. Genes are what is passed on from our parents in the form of DNA. Both parents contribute to giving a random combination of 50% of their genes. The cumulation of genes that you inherit from your parents will determine not only what you will look like but also what you are capable of achieving. Keeping in mind that parents also inherit genes from their parents, there may be certain genes in you that come from a linage of ancestors.
Why are genes so important? Genes in our bodies play a crucial role in our health. If we have a defective gene it could result in diseases such as cancer. A gene could also mutate or go missing, all these will greatly affect the wellbeing, health, and performance of the individual. According to FDA.gov:
"Sometimes the whole or part of a gene is defective or missing from birth, or a gene can change or mutate during adult life. Any of these variations can disrupt how proteins are made, which can contribute to health problems or diseases."
But what constitutes a genetic disease? Well, pretty much any disease passed down from your ancestors or a random mutation in your genes. Mutations are also referred to as copying errors within the DNA. let's look at an example of a mutation; Sickle cell disease. The condition roughly affects more than a hundred thousand people in the USA alone. The cause? a mutation that twists red blood cells into sickle-shaped crescents causing excruciating pain as they get stuck in blood vessels. People who have suffered from this disease describe the sensation as broken chards of glass flowing through their veins.
Enter Gene Therapy: In gene therapy, scientists are able to replace one gene with a medical problem with another that doesn't have one, turn off genes that are causing problems or insert genes that help the body fight or cure the disease. Genes are added by a vehicle called vectors, they are genetically engineered to transport the genes. Another promising technology; CRISPR is also a powerful tool by which scientists can modify genes. The new technology enables the modification of live cells, switch harmful genes off, turn beneficial genes on and edit particular DNA sequences. According to Scientific American, this rousingly popular term emerged about five decades ago! In fact, the concept was the main idea behind the film GATTACA (1997).
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| Figure2: GATTACA poster by Mason Kaye link |
The film takes place in the distant future, where the main character fantasizes about going into outer space, however, he struggles to fulfill that dream as he is a genetically inferior man in an era of designer humans. Before his birth, Vincent's parents didn't find it necessary to modify his genes. They then find out his life expectancy and a certain heart condition that stands as an obstacle between him and his dream. So Vincent was an inferior suitable for menial jobs, he was regarded as an invalid. His brother on the other hand reaped the benefits of gene therapy and did not have to worry about his health. Nevertheless, Vincent persisted, taking the identity of another man who was valid. The film shows the challenges he faces as he tries to reach the stars with someone else's identity, despite the many times his body gave him a hard time.
The film showed scenes of how he would compete with his brother and would use every ounce of energy he had in him to win. despite his physical limitations, he was able to achieve his dream. Figure 3 illustrates what he said when he defeated his brother in the last swim race he had with him.
Arguably, If vincent had undergone gene therapy he wouldn't have to face so many obstacles and go so far as to change his identity to achieve his dream.
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Figure 3: Vincent to his brother while competing in swimming.
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How far are we into this future? Initially, Gene editing in the early passage of time was very expensive and time-consuming, but the new CRISPR technology changed everything! It reduced the majority of the costs and the time needed. It also is something that can be practiced by anyone with its knowledge and a lab.
CAS9: When phages hunt bacteria in the ocean, in the rare cases that the bacteria survive, it is able to activate one of its most effective anti-virus systems. According to Kurzgesagt, after the attack the bacteria save part of the virus DNA in its own genetic code, this is where CRISPR comes in. The bacterium stores a copy from the archive, this acts as a defense weapon. Also regarded as a protein called CAS9. The protein acts as a scanner detecting the bacterium for a similar virus, which is then cut out. CAS9 is also programmable making it possible to edit genes preventing and even curing genetic diseases.
Kurzgesagt also highlighted how In 2015, scientists were able to get rid of HIV virus out of living cells from patients, This was a milestone that proved the possibility of the procedure. A year later they carried a larger experiment with rats who had the HIV virus in all their cells. 50% of the virus was removed from the body after CRISPR was injected into the rats tails. This highlights how only within a few decades CRISPR could help cure HIV. This also entails that CRISPR may give us the means to defeat viruses like herpes or diseases such as cancer.
When our cells refuse to die, while multiplying and concealing themselves from the immune system, we are experiencing cancer. To fight cancer, CRISPR would have to modify immune cells to fight signs of cancer within the body. Kurzgesagt describes it as " Getting a few injections of a few thousands of our own cells could help us treat cancer." USA approved clinical trial in 2016, while China went a step further declaring that they would be able to treat lung cancer patients with CRISPR in the same year. The potential of this technology could help us cure or prevent over 3000 diseases that could happen as a result of an incorrect letter in our DNA. According to Kurzgesagt this may take a decade or two. However, these treatments would be limited to the individual and will die with them unless they are used on reproductive cells.
This helps show how we are already living in a world where pre-selecting is normal and may advance to a higher level in the future. However, this future isn't coming anytime soon because CRISPR is not flawless just yet. Scientists fear that wrong edits may take place, the gene modification may be able to help achieve the desired result but there's no guaranteeing it won't trigger unwanted changes.
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