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Could Genetic Engineering be a way forward for treating genetic diseases?

Zoe Huang

Rules govern almost every part of our lives, controlling our every move, and overpowering each part of our body, including our cells. What if there was a way to change the rules? That is where CRISPR technology comes in. CRISPR is an extremely precise, genetic engineering technique that is revolutionising many aspects of medical care. Although still in its early development and research phase, observable benefits already exist.

The discovery of a mysterious palindromic, repeated DNA sequence in E. Coli was carried out in 1987. Since then, scientists have begun exploring and investigating the function of the peculiar phenomenon. This marks the start of a revolution. A new way to modify DNA, CRISPR technology was born. Gene editing may provide beneficial genetic changes that would otherwise take large amounts of evolutionary time. At this early stage, speculations about CRISPR's impacts seem infinite. What are the impacts of this new technology? How much will humanity change as a result of this? Precise genome engineering brings the potential to enhance things such as food production and medicinal discoveries. Research conducted over the last few years shows that CRISPR technology has the potential to alter crop resistance to disease and infection.

The inspiration behind CRISPR was found through observing nature, as were numerous advances in science and medicine. The idea was adapted from a defence mechanism found in bacteria and other microbes. To shield themselves from invasive pathogens, such microbes capture segments of the pathogen's DNA and store them as segments called CRISPRs or clusters, consistently interspersed with short palindromic repeats. From here onwards, stored DNA segments are converted into short pieces of RNA, assisting an enzyme called Cas, to seek and slice the invader's DNA. After this system was discovered, scientists realised that it had provided the foundation for the development of a versatile gene-editing tool, soon to be called CRISPR. In the laboratory, CRISPR is composed of two main components, a guide RNA, and a DNA-cutting enzyme, typically called Cas9.

In 2013, a game-changer occurred when researchers showed that CRISPR, a genetic engineering tool, had the ability to alter the DNA of human cells and could bring a revolutionary change in disease research and treatment. This method of gene editing has astonishing potential ranging from genetic disease treatment to modifying crops. CRISPR may be a future treatment for sickle cell disease, a genetic disease. Sickle cell disease is a consequence of a genetic mutation, making it ideal to treat using a CRISPR-mediated gene therapy. This treatment involves what is known as a gene-edited cell therapy procedure. During this procedure, hematopoietic stem cells are removed from the patient, corrected using CRISPR technology, and then replaced. Furthermore, CRISPR can also be utilised for cancer treatment. Since scientists concluded that changes in DNA lead to cancer, the search for correction methods began.

Although CRISPR has started a wave of enthusiasm, positivity, and support, as with many revolutionising discoveries, it has also attracted controversy and criticism. While it has great potential to be life-changing, CRISPR has raised a range of ethical concerns. For example, He Jiankui, a Chinese scientist spent three years in jail in 2019 following the announcement that he modified the DNA of newborn twin girls using the gene-editing tool, CRISPR. Ethical concerns concerning CRISPR are echoed by Claes Gustafsson, secretary of the Nobel committee in Chemistry and a professor of Biochemistry and Biophysics at Stockholm University stated that with "every really powerful technology in life sciences or everywhere, there's a possibility of misuse". Additionally, the development of CRISPR technology presents significant safety concerns. This is due to a risk of undesirable side effects due to off-target or flawed mutations. For example, an accidental modification of an oncogene or a tumour suppressor gene can lead to cancer.

CRISPR, a newly developed technique used for genetic engineering, has attracted attention over the years. It is proven to have numerous benefits towards an individual's health, ultimately improving their quality of life. However, significant ethical and safety concerns have also risen to the surface since the introduction of CRISPR. Is it really worth the risk? What are the long-term impacts of this? Do the benefits of CRISPR truly outweigh its limitations and the danger it could potentially cause to one's health? Some agree whilst others do not. So far, the role CRISPR has in our society is still in its infancy. What will happen in the future?

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