Genetic editing, designer babies, lab-grown organs – these medical breakthroughs seem like they came from a science fiction novel, and yet, they are being brought to life in labs across the world today. If you have heard of these things, chances are you have also heard of CRISPR or CRISPR-cas9, which is a revolutionary gene-editing tool that allows scientists to modify, remove and replace an organism’s DNA. According to Mrs. Marateo-Gonzalez, “An understanding of emerging gene-editing technologies will be important in the future. These technologies will be more common and individuals need that understanding to make informed decisions for themselves and loved ones”.
If you don’t recall from your biology class, DNA is the building block of life, it is the literal blueprint for how everything in your body functions and how it is created in the first place. The ability to edit DNA grants researchers the power to create and manipulate life itself. According to Medline Plus, compared to other gene editing tools of the past, such as TALENs or zinc-finger nucleases, CRISPR-cas9 is faster, easier to execute and replicate, cheaper, and it’s more precise than ever before. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are repeating DNA sequences that appear in bacteria. In fact, in certain bacteria, these “CRISPRs” act as an immune system that defends the bacteria against any antimicrobials that want to kill them. “CRISPR” is the main component of this genetic editing tool’s name because CRISPR-cas9 was based on the function of naturally occurring CRISPR’s in bacteria. Cas-9 is a naturally occurring enzyme also found in bacteria that acts like a molecular pair of scissors that specifically cut DNA.
Here’s how it works.
Cleveland Clinic explains that scientists will design a short “guide” RNA (gRNA) that matches with the DNA sequence they want to edit. This gRNA forms a complex with the Cas-9 enzyme, which uses the gRNA as a guide, or a kind of GPS, to locate the exact gene the scientists want to target. Once the Cas-9 arrives at the gene it cuts the DNA sequence, allowing scientists to delete, replace or repair the gene.
Why should we care?
If someone has a genetic mutation that is making them chronically ill, such as cell anemia, HIV, cystic fibrosis, sickle cell, or muscular dystrophy, scientists can use CRISPR-cas9 to correct the genetic mutation, eliminating the illness altogether within the patient’s body. CRIPSR-cas9 also has the capability to revolutionize cancer therapy by engineering the patients immune cells to be better at recognizing and destroying elusive cancer cells. It has the potential to revolutionize drug development by creating cell models to test new drug therapies in a faster and safer way that does not put human or animal lives at risk. However, as described by the National Institute of Health, genetic engineering will allow scientists to literally manipulate the foundations of life, and this brings about an ongoing debate about ethics in genetic editing. When asked about their opinions on the ethical implications of such a tool, Iris Pascal, Indian Hills junior claims, “Genetically yes, I think it is ethical to find cures but if you’re using it to alter IQ or appearance, I think it is unethical – especially since it is so expensive, which isn’ t fair for people from a lower socioeconomic class”. Similarly, Mrs. Marateo-Gonzalez claims that she, “would like to think that the scientists in our current society put more stock in the moral and ethical implications of their research but to quote Jeff Goldblum’s character Dr. Ian Malcolm from Jurassic Park, ‘Your scientists were so preoccupied with whether or not they could, they didn’t stop to think if they should’”.
CRISPR-cas9 is actively transforming incurable diseases into illnesses that are both treatable and preventable. This is not something that may happen in the future, it is happening now – right now, researchers across the world are conducting ongoing human clinical trials to turn CRISPR-cas9’s immense potential into real, tangible treatments for patients.