Blog 7: Curing Genetic Diseases Using CRISPR
Hey everybody. With AP Exams coming up I haven’t been able to put as much time into these blogs as I have in previous weeks so bear with me. After May 16th I’ll be done with all my exams and ready to start with denser blogs for you all. Anyway, for today’s blog I wanted to touch back into CRISPR and dive into how we are able to use it to cure Genetic Diseases.
I’ll walk you through how CRISPR is already curing many real genetic diseases and what this technology means for the future of medicine.
What are Genetic Diseases + CRISPR’s Importance?
I’ve touched on genetics a little in previous blogs but before I get started, let me run to give you some short contextualization. Genetic diseases happen when a person’s DNA mutates and causes health problems. Even a single wrong letter (A, T, C, G) can cause damages like blood cells breaking down, weaker muscles, or in more severe cases, disappearing vision.
Because the mutations with genetic diseases are in every single cell, traditional treatments of them can only manage the symptoms. They aren’t able to break it down and fix the root of the illness.
This is where CRISPR comes in.
How CRISPR is Curing Genetic Diseases:
Like I’ve mentioned in my last blog post, CRISPR gives scientists a way to go directly into a patient’s DNA and fix the problem from its root. However, here’s how the process of CRISPR is applied to genetic diseases specifically.
First, scientists collect some of the patient’s stem cells from either their blood or bone marrow. Once the cells have been gathered, they use CRISPR-Cas9 (see last post for definition) to carefully cut the DNA in the exact spot where the mutation is happening. After the DNA gets cut, the cell’s natural repair system activates and starts repairing the damages done by Cas9. With help from humans, the repair process can insert a corrected version of the gene into the place where the mutation was, essentially fixing the problem.
Once the cells get edited, they are put back into the patient's body where they can grow and create healthy cells to take over the mutated ones. Something that’s important to realize is that stem cells make copies of themselves. This means that if scientists were to fix even a small group of mutated cells, they could, realistically, completely rid a person of their genetic mutations.
Instead of just treating symptoms like traditional methods have done, CRISPR gives scientists a real chance to heal people at their genetic levels.
Example of CRISPR’s Impact: Now that you know what CRISPR is from my last blog, why it's important, and how it works in curing genetic diseases, I’ll cover a real life example where this technology has been applied and its impact.
Victoria Gray(sickle cell disease)
Victoria Gray was a mother of four children from Forest, Mississippi, who was diagnosed with sickle cell disease at three months old. Just as a reminder, sickle cell disease is a hereditary blood disorder that causes red blood cells to assume a sickle shape. This disorder leads to blockages in the blood flow, severe pain episodes, organ damage, and for many, a reduced life expectancy. So, to no surprise, Victoria suffered through constant hospitalizations. Her condition limited her day-to-day activities and decreased her quality of life for her entire life until she ran into CRISPR.
In 2019, Victoria became the first patient in the United States to undergo a CRISPR-based therapy for sickle cell disease. To do this, doctors first extracted her bone marrow stem cells and then used Cas9 to edit the gene. Using Cas9, they prompted her cells to produce fetal hemoglobin–a form of hemoglobin that prevents the sickling of red blood cells. After reinfusing the improved cells into her body, Victoria began to experience remarkable changes: she no longer went through pain crises, no longer relied on pain medications, and could even engage in her daily activities without any limitations.
Why is this such a Big Deal? Victoria Gray’s comeback story using CRISPR gene editing isn’t just luck but proof that this technology can really do something for medicine that's never been done before: cure a genetic disease from its root. In fact, by December 2023, the U.S Food and Drug Administration approved the CRISPR-based therapy. This approval marks a huge achievement for the gene-editing environment. With this technology, more people like Victoria Gray can finally heal and end lifelong complications.
What’s even more important to mention is that this decision by the U.S. The Food and Drug Administration opens the door for other genetic conditions to be cured too, not just the sickle cell disease I mentioned with Victoria. Diseases like beta-thalassemia, muscular dystrophy, and even some forms of blindness have begun to be tested for CRISPR-based treatments. It has made a massive impact and will only continue to grow in times to come.
Conclusion Before I close out today’s blog, I wanted to mention that CRISPR is still in its earlier stages and can have complications. In other words, not everything is as perfect and as simple as I make it out to be in the paragraphs above. However, what is true about this technology is that it gives us a chance at curing genetic diseases, not just treating their symptoms. I hope the story of Victoria Gray gives you a taste of how powerful this technology is.
As more clinical trials continue and the instruments get even better, CRISPR could offer real cures for diseases that we once considered “untouchable”. With the FDA’s approval, CRISPR’s revolutionary impact has just begun.
Thanks for reading, until next time!
— Aidan Kincaid
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