The University of Southampton

Author: Amelia Pennell

CRISPR-Cas9: A cure or a threat?

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Contents

Copy. Delete. Paste. Three words we all subconsciously think as we comb through text during our daily lives. Three words that I’ve been repeating endlessly as I spend countless hours cutting and pasting lines of code, desperately trying to make my third-year university project work. Combining the realisation of what an invaluable yet simple tool we have everyday access to and my studies in Biomedical Engineering, I began wondering if we could apply a similar gadget to our own DNA, removing any sequences we deem “undesirable” and replacing them with something of our choosing.

Available: https://stock.adobe.com/uk/search?k=paste+icon (Accessed: 23/03/25)

This led me to the discovery of Clustered Regularly Interspaced Short Palindromic Repeats, also known as CRISPR, which allows us to do exactly that, opening up a world of opportunities to cure disease as well as further the abilities of other biotechnologies – you can read more about the potential of a fascinating combination of stem cells and CRISPR-Cas9 here!

How does it work?

See below a brief video which explains how CRISPR-Cas9 is capable of editing our DNA!

Transcript: In a document, if we suspect we’ve misspelled a word we can use the find function to highlight the error and correct it or delete it. Within our DNA that function is taken on by a system called CRISPR/Cas9. CRISPR is short for clustered regularly interspaced short palindromic repeats. CRISPR consists of two components – the Cas9 protein that can cut DNA and a guide RNA that can recognise the sequence of DNA to be edited. To use CRISPR/Cas9, scientists first identify the sequence of the human genome that’s causing a health problem. Then they create a specific guide RNA to recognise that particular stretch of a’s, t’s, g’s and c’s in the DNA. The guide RNA is attached to the DNA cutting enzyme Cas9 and then this complex is introduced to the target cells. It locates the target letter sequence and cuts the DNA at that point. Scientists can then edit the existing genome by either modifying, deleting or inserting new sequences, effectively making CRISPR/Cas9 a cut-and-paste tool for DNA editing. In the future, scientists hope to use CRISPR/Cas9 to develop critical advances in patient care or even cure lifelong inherited diseases.

How could it be used?

One potential application of CRISPR-Cas9 currently being investigated surrounds sickle cell anaemia, an incurable genetic disease with only expensive and harmful treatments available. The potential to undergo a singular procedure to completely cure this condition is revolutionary – a potential that could be applied to up to 8,000 more genetic mutations.

However, the capacity for this technology is so great that I find myself beginning to fear what it could be used to eradicate instead of simply treat. Concerns are already being raised by scholars with genetic differences, statements such as “our genetic conditions are not simply entities that can be clipped away from us as if they were some kind of a misspelled word or an awkward sentence in a document” being published in scientific news journals, highlighting that someone is still human despite their differences. The desire to completely remove a gene from society assumes that people with such genes are constantly suffering, their gene pool contaminated and inherently inferior.

Personally, I carry the genetic mutation for haemochromatosis, a condition that means I will most likely be subject to regular venesection in my later adult years. Whilst I have no affinity for my condition, viewing it as separate to myself and something that I would readily “delete”, having access to the support groups has shown me how it can bring people together and create a beautiful community – something that can make some feel positively connected to their condition. The idea that we could use CRISPR-Cas9 to not only treat genetic diseases but instead completely remove them from existence raises the question of whether this technology is a cure or a threat to these communities.

Ethical Parallels

A parallel can easily be drawn between the ethical issues surrounding the application of CRISPR-Cas9 in curing instead of treating genetic diseases and those restricting gene editing on embryos. As of March 2025, it is illegal to perform gene editing on embryos for reproduction in the UK, “designer babies” being labelled as a “ethical horrors waiting to happen” by news companies as profound as The Guardian.

Available: https://asm.org/articles/cultures-magazine/volume-4,-issue-4-2017/the-designer-baby-distraction (Accessed: 23/03/25)

 

As a society we must be careful as we toe the line between providing the best quality of life and removing people’s individuality, a line that could easily be crossed by both of these technologies. In the end, I struggle to distinguish the difference between editing an embryo’s genes to create what is considered an “ideal” baby, a process that is currently illegal, and “perfecting” the genes that somebody already lives with. Despite this, I also wonder whether it is truly ethical to leave somebody wishing for a cure when one is sat right within our reach.

But in the end, how would you feel if a fundamental part of what made you you could be so easily erased from existence?

Type 1 Diabetes: The fight for a cure

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Imagine if your ability to eat relied on whether you could solve a handful of maths questions, lines of algebra blocking you from simply reaching over to the snack drawer and grabbing a bag of your favourite crisps. Sounds like a nightmare, right? But for 464,000 people in the UK alone, this is a version of their reality whilst living with type 1 diabetes, an autoimmune disease that prevents your body from producing insulin [1].

What is insulin?

Insulin, a vital hormone produced by the pancreas after the ingestion of food, enables our body to transfer glucose from our blood into our cells, allowing it to be used as energy [2].

Image available at: https://gluroo.com/blog/diabetes-101/insulin-faqs-type-1-diabetes/ (accessed: 09/03/25)

Those with type 1 diabetes have a pancreas that is unable to produce enough insulin to promote this transfer causing their levels of blood glucose to slowly increase with each meal, the sugars predominantly being expelled only as waste via frequent urination. As a result, insulin must be supplemented through injection, quantities being meticulously calculated dependent on what is being eaten and when. Whilst with years of practice calculating the amount of insulin you need can become almost second nature, many with this disease long for a day where they can eat freely, not having to constantly monitor their blood sugar levels and symptoms.

A short video explaining some key aspects of type 1 diabetes – closed captions are available!

Current Technologies

Whilst technology currently exists which aims to make the lives of those with diabetes easier, nothing works quite as well as a pancreas. For example, DexCom is a company which produces a small sensor which allows for the continuous monitoring of blood sugar levels, a connecting app receiving and displaying the statistics on a regular basis. Having an error of only 8.2%, this sensor would appear to be amazing in almost any other application, but when it comes to someone’s health you can never be accurate enough [3].

This video follows a young woman called Jordan and how DexCom allowed her to regain control of her life and love for sport (closed captions available)

With pancreas transplants carrying the same risks as any other major surgery, the possibility of rejection urging healthcare professionals to encourage the majority to manage their condition through diet and medication, most are left with these sensors as their most advanced form of care. Therefore, although some aspects of life with type 1 diabetes can be made more “convenient”, the fight for an accessible cure is still ongoing.

Stem Cells and Ethics

Excitingly, it has been reported that a 25-year-old woman with type 1 diabetes has undergone a stem cell transplant enabling her pancreas to begin producing insulin again, it continuing to do so even a year post transplant – more about this encouraging story can be read here! Whilst this seems promising, the procedure does come with risks. It is believed that a form of immunosuppression is required in order to prevent complications, making the patient more vulnerable to other illnesses and infections.

Furthermore, there is currently a large volume of debate over the ethics of using stem cells in treatments. Embryonic stem cells, those that are typically used in such treatments, can only be obtained through the destruction of a human embryo, a process that breeds concern surrounding the sanctity and rights of early human life [4].

Image available at: https://bioinformant.com/what-are-stem-cells/ (accessed: 09/03/25)

Whilst many believe using a potential human life in this manner is unethical, I can’t help but wonder if denying somebody a better quality of life that could be so easily provided is just as cruel. How would you feel if a cure to something that controlled your daily life was just out of reach?

References

[1]Diabetes UK, “How Many People in the UK Have diabetes?,” Diabetes UK, 2024. https://www.diabetes.org.uk/about-us/about-the-charity/our-strategy/statistics

[2] NHS , “What is type 1 diabetes?,” nhs.uk, Nov. 31, 2024. https://www.nhs.uk/conditions/type-1-diabetes/what-is-type-1-diabetes/

[3]S. K. Garg et al., “Accuracy and Safety of Dexcom G7 Continuous Glucose Monitoring in Adults with Diabetes,” Diabetes Technology & Therapeutics, vol. 24, no. 6, pp. 373–380, Jun. 2022, doi: https://doi.org/10.1089/dia.2022.0011.

[4] L. A. Cona, “Stem Cell Research Controversy: A Deep Dive (2023),” www.dvcstem.com, Sep. 14, 2023. https://www.dvcstem.com/post/stem-cell-research-controversy

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