Authored by Jacob Szlechter, Biological Sciences ‘24
The rapid rise in popularity seen within the field of biomedical engineering has led to countless technological breakthroughs since the turn of the century. Innovations such as the bionic exoskeleton, the camera pill, and various other biomedical advancements have revolutionized modern medicine. One of these technologies, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), has the potential to impact humanity on the largest scale. Simply stated, CRISPR technology allows for DNA editing at a nucleotide level, thus altering genes. CRISPR technology has created a massive buzz in the scientific community as it is both faster and more accurate than previous techniques of gene editing. It’s the most simple, versatile, and precise method of genetic manipulation to date. 
The genetic alteration process of CRISPR works by utilizing the Cas-9 enzyme and a guide RNA molecule (gRNA). The Cas-9 enzyme is directed to a specific location within the DNA by the gRNA, a small RNA sequence. At this location, the Cas-9 enzyme can make a double-stranded cut across the DNA resulting in edits at the break site. Upon recognizing the presence of DNA damage, the cell sends signals for repair. At this point, scientists can exploit this DNA repair machinery to introduce changes to one or more genes in the cell and gene of interest. 
In 2020, Biochemists Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in Chemistry for inventing CRISPR technology. In a press release following the honor, The Royal Swedish Academy of Sciences detailed that “Emmanuelle Charpentier and Jennifer A. Doudna have discovered one of the gene technology’s sharpest tools: the CRISPR/Cas9 scissors… this technology has had a revolutionary impact on the life sciences, (plus) is contributing to new cancer therapies and may make the dream of curing inherited diseases come true” . The overwhelming excitement surrounding CRISPR technology stems from the now feasible possibilities in combating life-threatening genetic conditions.
One of the genetic conditions that CRISPR technology strives to eliminate is sickle cell disease. Sickle cell disease is a genetic condition caused by the inheritance of two recessive genes that encode abnormal hemoglobin. The red blood cells that carry hemoglobin transform from a round disc shape into a crescent – or sickle – shape, compromising their ability to carry oxygen to the body. The challenge with this disease is that it causes a consistent shortage of red blood cells, as sickle cells have a shorter lifespan. Furthermore, sickle cells tend to block blood flow in affected patients which can lead to a variety of medical complications such as pain, infection, and stroke .
In 2016, a sickle cell patient by the name of Victoria Gray became the first person to be treated with CRISPR gene editing . The treatment of Gray’s condition was straightforward: her doctor first removed many stem cells from her body and genetically modified them with CRISPR. These edited cells were then placed back into Gray’s body to mitigate her condition. “The hope was the edited cells would produce a protein known as fetal hemoglobin, alleviating the symptoms of sickle cell” . After undergoing treatment, Gray’s life had been completely revolutionized. In an interview following treatment, Gray exclaimed, “I’m doing great. I don’t have any problems with sickle cell at all.” Since Gray’s breakthrough, medical professionals have treated “at least 45 patients with sickle cell and a related condition known as beta-thalassemia, and reported data indicating it’s working for at least 22 of them.” 
Though CRISPR technologies are a relatively new phenomenon, due to the incredible results of CRISPR gene editing to combat cancer and sickle cell disease, the scientific community believes that CRISPR will ultimately become an essential tool to combat a wide range of diseases. Ross Wilson, a colleague of Doudna at UC Berkeley who also works with CRISPR, had an optimistic outlook when asked about the future capabilities of CRISPR Technologies, professing, “our efforts will have a ripple effect to enable cures for blood disorders in general, like beta-thalassemia, as well as diseases of the immune system” .
Distinguished American author Walter Isaacson recently published a book titled “Codebreaker,” in which Isaacson discusses the importance of CRISPR to the future of the human race. In the novel, Isaacson delves into his belief that CRISPR will ultimately develop to a point where we can effectively edit every cell in the human body. If editing took place amongst early-stage embryos or in reproductive cells, the multiplication of these cells would ensure that the edited cells would dramatically alter the genome of the affected individual. In addition to affecting the edited embryo, these edits would also be passed down to subsequent generations. Isaacson states that once this technology becomes mainstream, it will not be long before our descendants, affected by CRISPR gene editing will essentially be an “edited human race” . Will CRISPR someday develop into a technology that will remarkably modify the human race? Only time will tell…
Your Genome. (2022, February 8). What is CRISPR-Cas9? Yourgenome.org. https://www.yourgenome.org/facts/what-is-crispr-cas9/
The Royal Swedish Academy of Sciences. (2020, October 7). The Nobel Prize in Chemistry 2020. NobelPrize.org. https://www.nobelprize.org/prizes/chemistry/2020/press-release/
CDC. (2022, August 18). What is Sickle Cell Disease? | CDC. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/sicklecell/facts.html#:~:text=SCD%20is%20a%20
Stein, R. (2021, December 31). First sickle cell patient treated with CRISPR gene-editing still thriving. NPR.org. https://www.npr.org/sections/health-shots/2021/12/31/1067400512/first-sickle-cell-patient-treated-with-crispr-gene-editing-still-thriving
Sanders, R. (2021, March 30). FDA approves first test of CRISPR to correct genetic defect causing sickle cell disease. Berkeley News. https://news.berkeley.edu/2021/03/30/fda-approves-first-test-of-crispr-to-correct-genetic-defect-causing-sickle-cell-disease/
Walter Isaacson Hopes CRISPR Gene Editing Will Be Used To Create A Utopia, Not A Dystopia. (n.d.). Www.youtube.com. Retrieved March 24, 2023, from https://www.youtube.com/watch?v=yZviX6VjaeY