The Code: How CRISPR is Rewriting the Future of Medicine

For a long time, having a genetic disorder felt like being born with a typo in your life’s instruction manual that cannot be erased. For example, if you were born with Sickle Cell Disease or Muscular Dystrophy, medicine would help you manage the symptoms, but it could not fix the source of it forever. That changed with the discovery of CRISPR-Cas9, a gene editing tool that acts as “molecular scissors” to cut that part of the gene.

What exactly is CRISPR?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), which was invented by Jennifer Doudna and Emmanuelle Charpentier, using the observations made in bacteria in 1987. Bacteria use this system as an immune response to fight off viruses by snipping pieces of the virus’s DNA to remember and disable it. 

Scientists figured out how to program to target specific sequences of human DNA from bacteria’s observations. By using a “guide RNA,” the Cas9 enzyme is led to a precise location in the genome. Once there, it makes a cut. The cell then tries to repair the cut, and during the process, scientists can remove a harmful gene or even paste a healthy version of the sequence. 

Real World Miracles: Sickle Cell Disease

Not just theory. In late 2023, the world saw the first regulatory approval of a CRISPR-based therapy for Sickle Cell Disease. Patients with this condition have “crescent moon” shaped red blood cells that get stuck in the blood vessels, causing extreme pain and organ damage. 

With this new treatment, doctors take the patient’s own stem cells, use CRISPR to edit the genetic instructions to produce healthy hemoglobin, and then infuse those cells back into the patient using a technique called myeloablative conditioning (high-dose chemotherapy). This is a process that removes the sickle cells from bone marrow and replaces them with the modified cells. The healthy hemoglobin produced in their bone marrow, once infused, is known as fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. If people with sickle cell disease have an increased amount of HbF prevent the sickling of red blood cells.

The Ethics of the “Red Line”

With great power comes a lot of debates, like the difference between Somatic editing and Germline editing. 

• Somatic: Involves fixing genes in a specific person’s cells, like their blood and lungs, to cure a disease. These changes are not passed down to their children, and are only used to cure the disease 

• Germline: Involves changing the DNA in embryos or reproductive cells. This means one can customize how their kid looks, and it would be inherited by future generations 

This is where the term “designer babies” comes from. While the idea of not having to worry about diseases forever is incredible, the scientific community is very cautious about it due to germline editing. If we can edit out a disease, we will eventually try to edit in more height, higher intelligence, or specific eye colors. Genomic experts agree that we should keep a firm line between medical necessity and “designer babies” so we don’t create deeper inequality based on genetic codes and their access at different places.

Looking Ahead

Genomic Medicine is no longer science fiction. We are moving toward a world where a “genetic defect” is simply a problem to be solved rather than a “life sentence”. As we continue to use CRISPR and refine it to make it more accurate, the possibilities of even treating blindness, heart disease, and certain types of HIV are starting to become possible.

Written By: Lakshmi Greeshma

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Works Cited 

Food and Drug Administration. “FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease.” FDA, 8 Dec. 2023, www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease.‌

Henderson, Hope, and Jodi Halpern. “CRISPR & Ethics.” Innovative Genomics Institute (IGI), Innovative Genomics Institute, 21 Nov. 2024, innovativegenomics.org/crisprpedia/crispr-ethics/.

“NIH-Funded Breakthrough Shrinks CRISPR for Precision Delivery in the Body.” National Institutes of Health (NIH), 13 Apr. 2026, www.nih.gov/news-events/news-releases/nih-funded-breakthrough-shrinks-crispr-precision-delivery-body.

‌Prasad K, George A, Ravi NS, Mohankumar KM. CRISPR/Cas-based gene editing: marking a new era in medical science. Mol Biol Rep. 2021 May;48(5):4879-4895. doi: 10.1007/s11033-021-06479-7. Epub 2021 Jun 18. PMID: 34143395; PMCID: PMC8212587.