CRISPR CAS-9

As innovations in biotechnology continue to change the future of medicine, it is 

evident that treatment options are evolving. The primary purpose of medicine is to 

treat a wide variety of diseases, including contagious, genetic, and developmental 

ones. Genetic diseases are hardest to cure, as it is challenging to alter, disable, or 

reactive specific genes to fight the diseases. UC Berkeley/UC San Francisco professor 

and Harvard Medical School Ph.D., Jennifer Doudna, discovered CRISPR-Cas 9 

(Clustered Regularly Interspaced Short Palindromic Repeats-associated with Protein 9). 

With the help of her colleagues, Doudna accidentally discovered this gene alteration 

technology while researching protein and virus interactions. CRISPR technology 

revolutionizes how scientists treat genetic diseases, as it allows medical professionals 

to edit genes more precisely and efficiently. Doing so prevents the disease from 

developing in the individual, thus eliminating the problem. Victoria Gray, a 34-year-old 

Sickle Cell patient from Mississippi, was the first person to receive treatment with 

CRISPR. Sickle Cell Disease is a devastating genetic disorder commonly found in people 

of African descent. A mutation in DNA causes a protein to form improperly, resulting in 

mutated hemoglobin. Hemoglobin is an essential compound that helps red blood cells 

carry oxygen and ensures the cell is round. If the hemoglobin is mutated, red blood 

cells become sickle-shaped, and they cannot carry enough oxygen. On top of this, the 

abnormal shape of the cell limits movement through blood vessels, especially small 

vessels. This increases the risk of developing a blood clot, which could lead to a stroke 

or heart attack. The average life expectancy of someone with Sickle Cell is around 40 

years. 

During a sickle cell crisis, blood clots begin forming in small vessels, restricting 

the flow of blood through the body. The patient may become anemic (have alarmingly 

low red blood cells and platelets count) and experience unfathomable pain. This can 

lead to feeling too weak to complete necessary tasks. Gray describes a sickle cell crisis 

as "horrible," especially when "you can't walk or lift up a spoon to feed yourself, it gets 

real hard." 

At the Sarah Cannon Institute in Nashville, Tennessee, Gray received an infusion 

of billions of cells that were genetically modified using CRISPR technology. The new 

cells boost protein production that alleviates pain during a sickle cell crisis. This 

drastically improves Gray's quality of life. Dr. Haydar Frangoul notes the "preliminary 

data shows for the first time that gene editing has actually helped a patient with sickle 

cell disease. This is definitely a huge deal." Vertex Pharmaceuticals from Boston and 

CRISPR Therapeutics from Cambridge, Massachusetts, continue to release data and 

sponsor the research in which Gray participated. Doctor Francis Collins, director of the 

National Institute of Health, states "gene therapy approaches" and "gene-editing 

protocol" are "the next phase of [healthcare]." Scientists and doctors are working 

towards applying CRISPR technology to treat cancers and beta-thalassemia. This 

indicates that CRISPR technology has a wide range of applications and can be used to 

treat virtually every genetic disorder; it has the potential to revolutionize healthcare. 

Alternative treatment options for sickle cell patients are available, most 

popularly bone marrow transplants. However, this process can be incredibly time- 

consuming, expensive, painful, and have a relatively high chance of failure. 

As of now, it is too early to determine if doctors can regularly use CRISPR 

technology to treat Sickle Cell patients. They will continue to monitor Gray and similar 

patients to determine if the treatment is effective and safe long-term. Despite these 

precautions, the application of this revolutionary medicine is significant, as it opens the 

door to the future of gene-editing and alternative treatment options in medicine. 

Victoria Gray is proof that the CRISPR technology is effective and helps patients 

recover, or at the very least, drastically improve quality of life. The future of treating 

genetic disorders continues to move towards using advanced versions of CRISPR, 

possibly the development of alternative gene-editing technology.

For more information please see:

https://www.npr.org/sections/health-shots/2019/11/19/780510277/gene-edited- 

supercells-make-progress-in-fight-against-sickle-cell-disease 

https://www.npr.org/sections/health-shots/2019/07/29/744826505/sickle-cell-patient- 

reveals-why-she-is-volunteering-for-landmark-gene-editing-st 

https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young- 

mississippi-womans-journey-through-a-pioneering-gene-editing-experiment 

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