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Biomedical Research

Gene Editing and Human Disease

As medical research continues to advance so does our understanding of gene editing, DNA, and human disease. But what are some of the implications of this advancement?

By Reem Hassoun

Published 6:33 PM EST, Mon February 22, 2021

Gene Editing: The Basics

While gene editing may sound like a sci-fi concept that is too futuristic to even think about implementing in the modern day, new improvements in technology have provided scientists with the means to not only make this happen now but to use it in order to improve the health of human beings all over the globe. Recently, gene editing as a whole can be narrowed down to one key technology—CRISPR, or clustered regularly interspaced short palindromic repeats. This remarkable tool gives scientists the power to alter the genome of a human, allowing them to replace, remove, and even change the location of the gene. CRISPR is based on bacteria and archaea, which are naturally found defense mechanisms against viruses that attack the body’s immune system, and it involves two main parts. The first part contains a series of DNA repeats, which work to store the DNA sequences that arrive from the viruses that attack the system. This allows the bacteria to “recognize” or find the DNA of the virus if it attempts to invade again. Additionally, this prevents the virus from doing damage by sending this DNA to the second part. This part involves the actual gene editing, where it cuts or alters the DNA of the virus that was sent through the repeat in the first part.

The Inner Workings of CRISPR Technology

Figure 1: Bernie Hobbs, CRISPR: The new tool in the gene editing revolution explained.

Understanding the Implications

With CRISPR, scientists are able to take these basic principles and apply them to utilize gene editing in the lab. However, with every new advancement comes both negative and positive implications. When it comes to the more negative side of gene editing, ethical concerns are often brought up and taken into account. For instance, gene-engineered human creation is very controversial amongst the gene editing community. With the more widespread implementation of technologies, including CRISPR, that make this possible, people are becoming wary of its potentially detrimental impact on human nature for future generations. Despite these concerns, the outlook for gene editing is predicted to have monumentally positive impacts on humankind in terms of long and short term development. One main factor that draws many scientists to CRISPR is its potential to cure and aid a myriad of human diseases. These include blood disorders, AIDS, Cystic Fibrosis, Huntington’s Disease, blindness, and even cancer. In fact, the Hangzhou Cancer Hospital is currently working on a clinical trial which removes the immune T cells from the patient and extracts the PD-1 encoding gene through cell modification done with CRISPR. The modified cells are then able to be placed back into the patient with a larger aptitude to attack the cancer cells. With this technology spreading rapidly, more scientists are able to conduct trials like this, which have the potential to save millions of lives and improve the standards of living for all individuals—especially those whose lives revolve around their sickness or the sickness of a loved one. At the start of the decade, this level of innovation wasn’t even thought to be possible. Now, CRISPR and gene editing advancements changed the entirety of the medical and scientific communities, providing scientists with a tool that has the ability to change the world for the better.

Reem Hassoun, Youth Medical Journal 2021

References

Clara Rodríguez Fernández – 23/07/2019 7 mins – CRISPR, Tops. “7 Diseases CRISPR Technology Could Cure.” Labiotech.eu, 14 Jan. 2020, http://www.labiotech.eu/crispr/crispr-technology-cure-disease/. 

“What Are Genome Editing and CRISPR-Cas9?: MedlinePlus Genetics.” MedlinePlus, U.S. National Library of Medicine, 18 Sept. 2020, medlineplus.gov/genetics/understanding/genomicresearch/genomeediting/. 

You, Liting, et al. “Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research.” Molecular Therapy. Methods & Clinical Development, American Society of Gene & Cell Therapy, 15 Mar. 2019, http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6447755/.

By Reem Hassoun

Reem Hassoun is a student at Dearborn High School in Dearborn, Michigan. She is interested in the fields of neurology, public health, and biochemistry.

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