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The Treatment of Our Age: CRISPR

We are in the age of technology where nearly any disease can be cured or precautioned by the usage of medicines and vaccines. However, the treatment of several illnesses regarding genes and the DNA sequence of cells still remains unfound.


Sickle cell was the first ever illness to be described as a molecular disease. This disease affected over 100,000 people in the United States and also many people in Africa, since this disease could be genetically inherited from parents to their children. Even though the gene that creates the sickle cell helps people in Africa defend their body from Malaria, the carrying of this gene causes problems due to contractions. Thus, a solution to this illness was severely required.


In 2012, it was mentioned by the researchers Jennifer Doudna and Emmanuelle Charpentier for the first time that new RNAs could be made to influence a Cas nuclease and could be used in any DNA sequence. Moreover, in 2020 the same researchers became the laureates of the Nobel Prize in Chemistry for discovering one of the most efficient gene editing technology tools, the CRISPR/Cas9 genetic scissors. Even though there were different technologies to cure these molecular diseases CRISPR made it much cheaper and easier, making it a better choice.


But how do these genetic scissors work? The word CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats'' and Cas stands for “CRISPR-associated”. With these technologies, it becomes easier to cure a contraction in a specific gene by changing the DNA sequence by adding a different series to modify. The process goes like this, firstly the CRISPR protein is inserted to a cell along with a guide RNA. After that, they hook onto each other and move along the DNA to find a 20-DNA-letter long serie that they could attach to. This process gets even more impressive and interesting when we consider the fact that the shortest strand of a single DNA involves nearly 50,000,000 nucleotides in length.


This technology does not only protect or save one from disease, but also it is believed that it can be used to change the genes of unborn children. In this case, ethical concerns come to mind. The danger this high technology poses for being able to be used for bioterrorism by anyone or changing the genomes of unborn children being believed to create issues in the following years when CRISPR/Cas9 becomes more popular and widely used. Since both of these risks can harm humanity severely with long-lasting effects that will affect the next generations badly as well, it is believed that it will pose perils in the area of physiology and medicine.


Let’s now talk about the declaration the United States made that says that they approve Casgevy and Lyfgenia. On the 8th of December the U.S Food and Drug Administration (FDA) accepted the usage of this new technology, CRISPR, for the treatment which will be done by using Casgevy, one of the first gene therapies for the treatment of sickle cell disease in people at the age of 12 and older, utilizing CRISPR/Cas9. The director of FDA’S Center For Biologics Evaluation and Research Dr. Nicole Verdun stated that: "Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited…”. With these sentences we can understand that the small number of types of cures for a wide range of illnesses led to a need for a brand new treatment option and CRISPR/Cas9 was the best option possible due to the opportunities it presents.


We do not know what is waiting for us in the upcoming years. With the improvements in the technology and the areas of medicine, chemistry and biology cures for even more diseases including cancer may be found. However, I personally believe that up until then the CRISPR/Cas9 treatment will be enhanced and the usage areas will be expanded so that it can be used as a way of rehabilitation for many more illnesses.


Works Cited

The Jackson Laboratory . “What Is CRISPR?” The Jackson Laboratory, 2012.

“Jennifer Doudna and Emmanuelle Charpentier Win 2020 Nobel Prize in Chemistry | UNESCO.”, UNESCO.

Peebles, Angelica. “U.S. Poised to Approve First Gene-Editing Treatment in Breakthrough for Sickle Cell Patients.” CNBC, 7 Dec. 2023.

Le Page, Michael. “What Is CRISPR?” New Scientist.

Brown, Terence A. The Human Genome, NCBI.

Caplan, A. L., et al. “No Time to Waste--the Ethical Challenges Created by CRISPR.” EMBO Reports, vol. 16, no. 11, 8 Oct. 2015, pp. 1421–1426.

Ayanoglu, Fatma Betul, et al. “Bioethical Issues in Genome Editing by the CRISPR/Cas9 Technology.” Turkish Journal of Biology, vol. 44, no. 2, 2 Apr. 2020.

“FDA Approves First CRISPR Treatment in U.S.” TIME, 8 Dec. 2023.

Commissioner, Office of the. “FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease.” FDA, 8 Dec. 2023.

“Matching CRISPR to the Job Improves the Safety, Efficiency of the Gene-Editing Tool.” UT News, 8 Sept. 2020.

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