When you think of CRISPR, the gene-editing tool, you might picture a dystopian future with designer babies, or perhaps a sci-fi scenario like Peter Parker gaining superpowers from a spider bite that alters his DNA. But in reality, CRISPR is far from either of those extremes (sorry to anyone hoping to become the next Spiderman). Instead, it's becoming one of the most promising breakthroughs in medicine, and it's all happening right now.

CRISPR was first discovered in bacteria and archaea as a naturally occurring defence mechanism against viral infection. In 2012, scientists Dr. Jennifer Doudna and Dr. Emmanuelle Charptentier realised that this system could be repurposed for “gene editing” in humans. In essence, CRISPR allows scientists to "cut and paste" DNA sequences, making targeted changes to the genome. Their breakthrough work awarded them the Nobel Prize in Chemistry in 2020.

The power of CRISPR in medicine lies in its ability to correct genetic defects at their source, rather than just managing the symptoms. As Dr. Siddhartha Mukherjee, a well-known oncologist and author of The Song of the Cell, said, "soon we’ll treat disease with a cell, not a pill.”

A recent advancement in CRISPR is its application in treating two genetic blood disorders, sickle cell disease and beta thalassemia. Both conditions are caused by mutations in the gene responsible for producing haemoglobin, the protein in red blood cells that carries oxygen. In sickle cell disease, the mutation causes red blood cells to become sickle-shaped, impairing their ability to transport oxygen and leading to severe complications like pain and fatigue. In beta thalassemia, the mutation leads to reduced haemoglobin production, resulting in low blood oxygen levels.

Thanks to CRISPR, scientists have developed a treatment that edits the DNA in blood stem cells, where the mutation of haemoglobin gene is found, allowing patients to produce healthy haemoglobin. This therapy doesn’t just alleviate symptoms — it offers potential to cure these diseases.

In December 2023, after less than a decade of CRISPRs discovery, the FDA approved the first-ever CRISPR-based gene therapy for sickle cell disease and beta thalassemia, marking a historic milestone in the field of medicine.

One of the first and most notable patients to benefit from CRISPR is a 15-year-old Jonathan Lubin, diagnosed with sickle cell anaemia since he was born. Like most with sickle cell disease, Jonathan struggled to engage in everyday activities due to the limited oxygen in his blood. Being in and out of hospitals for most of his life, Jonathan’s parents were in need of a solution and they took the chance with a clinical trial for a new technology. Following the trial, Jonathan described the experience as “cool and freaky.” The trial's results showed that 29 out of 30 patients have been free from the painful crises from sickle cell anaemia for a year, successfully reaching the clinical trials goals.

Jonathan Lubin (Age 16); Taken from Health Matters - NYP

The economic implications of CRISPR are also massive. Companies like CRISPR Therapeutics, which developed the gene therapy for the sickle cell anaemia and beta thalassemia, have seen a surge in investor interest. Analysts predict that the market for gene-editing technologies will explode in the coming years, as more therapies move from clinical trials to real-world applications. The ability to cure genetic diseases opens up new avenues for biotechnology firms, attracting billions in funding and reshaping the landscape of healthcare.

While CRISPR holds incredible promise, it also raises ethical questions. Should we use gene editing to prevent life-threatening diseases? What about enhancing traits in humans? These are debates we must engage in as CRISPR technology continues to evolve. Just as Peter Parker's new abilities came with both extraordinary gifts and heavy burdens, we too must consider the responsibilities that come with this powerful technology. Uncle Ben’s timeless words resonate here, “With great power comes great responsibility.”