CRISPR- The Future of Disease Treatment

Updated: Sep 22, 2019

What would you do if you had the chance to change your life through experimental medical treatment? Got your answer? Okay, now what if you were the first person to ever try this new technique? This was the same chance that sickle cell patient, Victoria Gray was given earlier this year.

Victoria Gray is a 41-year old woman from Mississippi who suffers from sickle cell anemia. Sickle cell anemia is a painful disease in which instead of your bone marrow making the protein beta-hemoglobin, which makes your blood cells nice and round, the marrow produces the defective protein, hemoglobin S, which gives the blood cells a sickle shape. These hook-shaped cells slow down the transport of oxygen throughout the body and often, the cells get stuck in the smaller blood vessels and breakdown, leaving many organs and tissues in the body without the oxygen they need. "It's horrible," Gray explained to NPR. "When you can't walk or lift up a spoon to feed yourself, it gets real hard”. The only known cure at the moment for the disease is a donor transplant that works for just 10 percent of patients affected by sickle cell anemia. However, this treatment, if successful, would be effective for all sickle cell patients. Now what exactly is the treatment?

CRISPR (pronounced “crisper”) is a powerful genome-editing technique that allows researchers to efficiently modify DNA sequences and gene function. It helps to repair gene defects and to prevent and treat spreading diseases. CRISPR is short for CRISPR (Cas9). CRISPRs, or “clusters of regularly interspaced short palindromic repeats” are specialized stretches of DNA, and the protein Cas9 is capable of cutting strands of DNA. This groundbreaking technology was adapted from the natural defense systems of unicellular microorganisms, such as bacteria. The first time that the world was really able to see the process of the CRISPR technique in action was in November, 2017, in a paper published by a team of researchers in Japan, led by Mikihiro Shibata and Hiroshi Nishimasu.

Gray, as well as her doctors, decided to try using CRISPR technology in hopes of editing cells extracted from Gray’s bone marrow and creating normal functioning red blood cells. This experiment was highly anticipated by researchers all around the world because Gray was the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease. After being infused with billions of genetically modified cells, it will most likely take months of Gray being closely monitored to know if the treatment is safe.

Most scientists agree that it is ethically ok to use CRISPR technology on adult cells. In fact, many researchers even compare the process to taking a pill. However, a much more heated debate arises when the topic of CRISPER technology on human embryos comes up. Last year, Chinese scientist He Jiankui used CRISPR to edit human embryos in attempts to disable the gene CCR5. In November of 2018, after having the edited embryos implanted, a woman gave birth to twin girls. The purpose of disabling the gene CCR5 was to make the embryos resistant to the HIV virus carried by their father. Even though this may sound like a good idea, according to hematologist, Vivian Sheehan, when you use CRISPR to edit an embryo, you are altering every cell in that person’s eventual body. So when Jiankui disabled the CCR5 gene, he disabled it in every cell in the girls’ body, which could have extremely dangerous and unknown side effects. Jiankui faced an enormous amount of criticism from researchers all over the world, as well as losing his job and may even be facing jail time.

Although there are some controversies surrounding CRISPR, if this treatment proves to be successful, it could mean a better quality of life for countless people globally.

By Cora Hafer


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