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Commentary

The Genetic Game Changer: CRISPR

10/29/2024

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By R. Renzo
Picture
CRISPR, a name frequently mentioned in the field of genetics, has been slowly proving itself as a groundbreaking tool in genetic editing. But what exactly is it? 
 
Although CRISPR is used to refer broadly to the systems used to target and edit DNA, the term stands for "Clustered Regularly Interspaced Short Palindromic Repeat". This makes reference to repeated repetitive DNA sequences in bacteria with "spacer" DNA sequences. When a virus infects a bacterium, the bacterium can capture and store small segments of the virus’s DNA, known as "spacers". In the natural genome (complete set of genetic material in a cell) editing processes inside bacterial cells, the cells transcribe the "spacer" sequences to RNA upon viral infection. RNA then has the capability of locating DNA sequences that match the "spacer". When the target material is found, one of the enzymes produced by the CRISPR system binds to the DNA and cuts it off, shutting off the gene.  

In a groundbreaking way, researchers have found ways to use CRISPR to enable gene activation
, and not its deletion. With these systems, we are now able to permanently modify genes in living cells and organisms, and possibly even make it possible to correct mutations at precise locations in the human genome.

 
As this technology continues to refine itself, the possibilities are endless. It's primary impact area seems to be in treating human diseases with a known mutation in the gene of cells. A rising treatment is that of sickle cell anaemia, a genetic blood disorder that affects the shape of red blood cells, making them look like sickles. These misshaped cells clog critical blood vessels, causing obstructions that can lead to severe pain, infections or even strokes. Treatment options for patients who suffer from sickle cell anaemia are currently limited. Patients require frequent blood transfusions, and their medications typically address only the symptoms that arise because of the disease, but not the root of it. Very recently, in December of 2023, the FDA approved the very first CRISPR-based therapy, where the main approach is to correct the haemoglobin gene mutation responsible for sickle cell trait, causing the healthy form of the gene to be produced. Currently, clinical trials are taking place in order for this treatment to be put into action. 
   
In addition to being able to edit the cells that make up most of the body, it is possible to edit the genetic material of gametes (eggs and sperm cells) as well as early embryos. This is called germline editing, and any such edits would affect the affected person's genetic makeup, as well as the ones that inherit the DNA. However, there are some ethical issues that arise with such a powerful tool – this technology could be used to theoretically improve the desirable traits instead of curing disease. For now, scientists have put aside germline editing because of its ethical and societal implications, but this may be something implemented in the future. 
 
Imagine being able to choose your own child's eye colour, height and even intelligence! CRISPR has already changed the game for the treatment of disease, but can it change the cosmetic industry as a whole by introducing customizability to foetuses? Will we ever live in a society where this is not stigmatized, and even turns normal? 
​

Bibliography
  • https://www.jax.org/personalized-medicine/precision-medicine-and-you/what-is-crispr 
  • https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/questions-and-answers-about-crispr  
  • https://www.synthego.com/crispr-sickle-cell-disease#:~:text=As%20sickle%20cell%20disease%20is,%2C%20corrected%2C%20and%20then%20replaced.  
 
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