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Genetics- The Future of Medicine

Everyone knows what genes are- they are the things that make you, you. To elaborate, genes are formed from the strands of DNA found at the center of the cell nucleus. “The Human Geonome Project has found 25,000 genes in human DNA,” according to Deane Alban (reset.me). A helpful analogy is thinking of your body as a recipe, and each gene is an ingredient. Each gene includes the “instructions” to create specific proteins in the body.  It’s these proteins that determine the structures and functions of each of the cells in the body, and that’s how genes dictate inherited characteristics. However, genes can change throughout your lifetime as a result of diet, environment, and more; these are called epigenetic changes. Apparently, certain lifestyle choices can trigger or turn off specific genes. So, in many ways, we aren’t entirely victim to our genetic makeup, which is a comforting thought. On the flip side, genetic mutations such as cystic fibrosis and color blindness are permanent; but again, various genetic mutations can be triggered through the environment and lifestyle choices (examples include radiation, cigarette smoke, etc). To reiterate, although we are in control of our genes for the most part, there are many aspects of our bodies inherited that are irreversible, and potentially fatal.

Now with some background on human genes, we can delve into how genetics is changing medicine. According to Washington Post, scientists have discovered a way to change genetic codes (“and the disease genes embedded in it”) after billions of years.

“Not only have we learned to read and write the genetic code; now we can put it in digital form and translate it back into synthesized life. In theory, that gives our species control over biological design. We can write DNA software, boot it up to a computer converter and create unlimited variations of the gene sequences of biological life.”

Although controversial, it’s been an incredible leap forward for medicine. After much research, this discovery has shown that many fatal diseases (ex. Huntington’s disease) are caused by one, single faulty gene. With this in mind, scientists set on a mission to find a way to create a genome for themselves. After many different attempts, they found a way to synthesize chromosomes, thus creating their own synthetic genome. This proves that it is possible to create genomes, and there is hope for helping the people that are victims to their genetic makeup. A big issue is cancer risk-and scientists are discovering ways to predict disease risk. (Of course, there were various other complex projects involved in their discovery of how to change genetic code; you can read more about it in this Washington Post article).

Function of the crispr cas9
Image from Vox

After discovering that many dangerous, genetically-caused diseases are the result of one defective gene, scientists were able to develop tools to help correct it. “Editing tools” like CRISPR performs gene therapy and ex vivo gene therapy. Scientists have run into some difficulty for the first kind of gene therapy- it is impossible to correct the genomes in the incredible number of cells that the human body possesses. However, ex vivo gene therapy- in which they correct the cells and then return them to the body- has been shown to work. As Craig Venter said, “There have been some encouraging results with cancer treatments using immune cells that have been edited with CRISPR. I think we will see even more success in cancer treatments using ex vivo gene editing.”

This gene therapy is nothing short of revolutionary. After billions of years of being victim to genetic makeups, scientists have found ways to prevent or cure diseases (and traits) that plague many across the globe. With CRISPR, scientists are able to remove harmful genes and add new ones.

Nevertheless, while it may seem like a miracle, gene therapy has become a heated topic. Why? To make sure that it works, scientists must perform human experiments to test whether it’s safe and effective. Tests are also being done to determine how to “cure” different types of diseases. And there’s more- these “editing tools” aren’t quite as meticulous as they seem, meaning that other genes may be altered, not just the targeted one. Most of the controversy revolves around the simple question- what if the scientists succeed? Many are against it because it’s “unnatural”- despite having the capability to cure dangerous diseases- and they are afraid that scientists could create a new race of superhumans.

“I am sure that during this century, people will discover how to modify both intelligence and instincts such as aggression. Laws will probably be passed against genetic engineering with humans. But some people won’t be able to resist the temptation to improve human characteristics, such as memory, resistance to disease and length of life. Once such superhumans appear, there will be significant political problems with unimproved humans, who won’t be able to compete. Presumably, they will die out, or become unimportant. Instead, there will be a race of self-designing beings who are improving at an ever-increasing rate,” states Stephen Hawkings.

To conclude, gene therapy is a great step for medicine. It’s a huge discovery- and while people haven’t decided whether it’s good or bad, it’s monumental. It seems that Newton’s Third Law of Physics applies- “For every action, there is an equal and opposite reaction.” Scientists found a lead on a way to fix genes and the deadly diseases they may cause, but must perform human testing to get there- potentially putting the very humans they wanted to save in danger. And if they do succeed, many diseases will be cured, many lives will be saved… but there’s the possibility of this new technology being used to create “superhumans” that humans will not be able to compete with (“they will die out, or become unimportant”).

Either way, genes are decidedly the future of medicine- and despite the many pros, there are, of course, cons- but this new branch of science will change medicine as we know it.

 Edited by: Karen Yung and Shreya Singireddy