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CRISPR-Cas9: A cure or a threat?

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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?

Gene Editing: Does it Hurt those it’s Meant to Help?

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When we first looked at gene editing, I had mixed feelings. As some who studies engineering, I believe in innovation and using technology to help people. However, as some whose sibling has a disability, I thought about how the advancement of gene editing pushes the narrative that those with disabilities need ‘fixing’. Therefore, I decided to research the topic further.

First of all, what is gene editing?

Gene editing is the process of deleting, inserting or replacing genetic material within animals, plants and bacteria to alter their characteristics. It has different applications, but I’m focusing on gene therapy and using different techniques to treat diseases. The development of CRISPR-Cas9 has created a quicker, cheaper method for gene editing, leading to the current buzz around the topic.

Laws surrounding gene editing:

While it is illegal in the UK to implant a gene-edited embryo, in 2016, the HFEA approved licensing to allow gene editing of human embryos in research. Many of my classmates thought this was a good change as it could lead to more knowledge and potential cures about inheritable disorders like Cystic Fibrosis. However, is this any different from previous attempts in eugenics? The removal of genetics at an embryonic level will lead to the eradication of different, ‘undesirable’, traits from society. The practice may also lead to the relaxation of laws and the possibility of designer babies.

Gene therapy case:

While gene editing is often associated with inheritable disorders, it can be used to cure cancer. There have been successful results from a study in 2010, where a patient suffering from lymphoma underwent CAR T cell therapy. In this treatment, the patients T cells are collected and then genetically altered in the laboratory so they can recognise the cancerous cells. They are then put back inside the body to fight the cancerous cells.

Image of CAR T-Cell Therapy

Cost of gene editing:

However, gene therapies are expensive! In the US it is estimated that $20.4 billion is spent annually on gene therapies. If this money was spent on creating a more inclusive environment through education of the public and the changing of laws, this could have a far greater effect on the people already living with genetic disorders. Is it not better to create an environment where people can live well with these disorders, then create one which focuses on their removal?

Different opinions:

The NHGRI conducted surveys to investigate patient perspectives on gene editing. Many patients, especially those with Huntington’s, argued that gene editing should be used to prevent other people from inheriting the disease, despite the argument that it could isolate them from society and reinforce the belief that people with disabilities have a low quality of life.

Furthermore, Wellcome Connecting Science hosted a citizen’s jury vote based on the following question: ‘Are there any circumstances under which a UK Government should consider changing the law to allow intentional genome editing of human embryos for serious genetic conditions?’. All the jurors had been affected in one way or another by hereditary diseases and by the end most jurors (17-4) agreed that human embryos should be edited. While a small sample, this vote indicates that the scientific community and the legislators are listening to those who it truly affects, something which has previously been overlooked and distinguishes gene editing from previous, eugenic practices.

Final Thoughts

My summary of different arguments for and against Gene-Editing

I believe that the advancement of gene editing will help those with genetic disorders and provides cures which were previously unavailable. I think that this outweighs the negatives of gene editing especially considering many people with genetic disorders believe in the benefits. However, I think that the narrative surrounding gene editing needs to include those who are affected the most to make sure that it is continuing to be done in a positive way which doesn’t isolate people or become modern-day eugenics.

fNIRs: The future of Cerebral Imaging

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Following the Sensor Lectures given by David Simpson and Russel Torah, the idea of optical imaging really stood out to me. Additionally, my passion for neuroscience and the brain brought me to cerebral optical imaging. Upon initial research, I noticed the most documented imaging techniques included MRI, CT and PET scans. PET scans, or positron emission tomography involves the use of a radio tracer that emits positively charged particles. When these particles encounter an electron, they completely annihilate and generate two gamma rays that can be picked up by the PET scanner. I had known about annihilation from physics at school, but I never knew to could be used to image the body! Since the radiotracer gets absorbed into more active tissues. This makes PET unique as it’s a way to image the functionality of tissues.

Looking closer into functional imaging, I came across functional MRI (fMRI) and fNIRs. While fMRI builds on current MRI technology, it involves the patient to stay still while completing tasks. While I knew this was still useful, I found that fNIRs could be used while moving around, so that, in my opinion it was much more potential in comparison.

fNIRs: A Quick Rundown

Functional near infrared spectroscopy, is a functional cerebral imaging method. It involves the use of near infrared light, between 700-950nm wavelengths. This is because once you get to the near infrared, the body is nearly transparent! Using a source, like an LED you pass the light through the body where the light follows a banana shape towards a detector just next to the source. I found this particularly strange, it made me think how light of all wavelengths of light pass through the body.

This is a short video on the underlying principles of fNIRs.
Advantages of fNIRsDisadvantages of fNIRs
High Temporal ResolutionLower Spatial Resolution
Non-invasive & safeSurface level measurements
PortabilitySensitivity to surface (i.e. hair, skin pigment)
Resistance to motion artifacts
Cost effective

Where are the images?

An real image of fNIRs/DOT technology

You may have noticed there aren’t any images being generated by fNIRs. This is because in order to generate images you must use Diffuse Optical Tomography (DOT). Simply put, DOT involves using lots of fNIRs sources and detectors and overlapping them to generate an image. Using these images in conjunction with a functional experiment, you can see which areas of the brain ‘light up’ when conducting particular activities. Since fNIRs can be used while moving, the sky is the limit when coming up with experiments to conduct.

The Promising Future of fNIRs/ Reflection

After discovering this promising technology, I felt compelled to write about it. I found its resistance to motion artifacts particularly interesting, as a biomedical engineer, they are something I have to deal with in all of my projects. Additionally, this feature means fNIRs has the potential to investigate infant brains as MRI and fMRI require the patient to stay very still, which can be very difficult for infants and children.

A researcher at the University of Southampton; Dr Ernesto Elias Vidal Rosas, is currently working on an fNIRs system that will tackle the issue of the lower spatial resolution, one of the main drawbacks of the technology. He inspired me to investigate this technology after discovering one of his written papers on the topic.

Personally I see this technology rivalling that of fMRI in its researching capabilities in infants not only due to the motion resistance but also the ability to conduct naturalistic experiments, potentially using technology like VR in order to investigate the brain’s activity when interacting with the world outside the lab. Additionally, the sensor could be used in other areas of the body for example, when placed just below the ribcage, “[fNIRs] has shown promise in being a more accurate, and less bias sensor compared to the gold standard”, Dr Ernesto Elias Vidal Rosas.

The Amazing Spider Goat

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Can they swing from a thread? No, but they can produce them!

Several years ago, in my GCSE biology class, I first encountered the concept of gene editing and transgenic animals. Since then, I have become very interested in medical devices and biomaterials. Spider goats perfectly combine these topics and pave the way for potentially revolutionary advances in medicine and healthcare.

Spider Goats

Spider goats appear to be completely regular goats at first glance. However, they have a secret superpower: the ability to produce the spider silk protein in their milk!

How are they made?

There are various ways to produce transgenic animals. The way it was originally achieved in spider goats was by taking some skin cells from the goat and inserting the gene from the spider that creates the silk protein into the nucleus. The nucleus could then be removed from the skin cell and put into an egg cell, which was then implanted into a goat. The result is a goat that can produce the protein for spider silk in its milk. [1]

How is the silk extracted?

In an article written by T. Miller [1], she interviews Justin A., who helped create the original herd of these spider goats, and he explains how the silk is extracted from the goat’s milk.

Firstly, the goat’s milk is collected and frozen. Once the milk has thawed, it can be purified. The first step is to remove any fats and then filter out the smaller proteins. The spider silk protein is separated by selective precipitation (separating a solid from a solution [2]) and washed. Once a purified form of the spider silk protein is obtained, it is suspended in water and placed in a microwave. The purified liquid silk protein can be easily manipulated into its desired form.

THE AMAZING SPIDER GOAT (a short comic)

Spider Silk

Its structure and properties

The type of spider silk focused on by most studies is dragline silk. It has some interesting properties, such as having a good balance between strength and elasticity to enable the spider to catch flying prey. These unique properties means that it surpasses the majority of all natural and man-made materials [3].

Some of its most beneficial properties [3]:

  • Strong
  • Tough
  • Biocompatible (compatible with living tissue [4])
  • Minimally immunogenic (produces an initial immune response, but there are no long term effects)
  • Exceptional cytocompatibility (has very little effect on the structure and function of tissues it comes into contact with [5])
  • Slow degradation rate

Did you know? Spider silk produced by transgenic silk worms has been found to be 6 times tougher than Kevlar, which is used in bullet proof vests! [6]

These properties are a result of mainly two spidroins (proteins) that comprise the silk: MaSp1 and MaSp2. These proteins have very repetitive amino acid sequences that lead to the formation of specific final structures that give the silk its unique physical properties.[3]

Potential medical uses of spider silk

The use of spider silk in medicine is certainly not a new idea. In fact, it was even used by the ancient Greeks and Romans to promote wound healing [3]!

The possibilities for the biomedical applications of spider silk is what particularly interests me about this topic, since it relates to the majority of the modules I am currently studying. For example, both this module and the Orthopaedic Biomechanics module have covered tissue engineering and spider silk has shown great promise to enhance current solutions in this area [7]:

  • Skin regeneration and wound dressing
    • It has been shown to promote the healing of burns
  • Cartilage and tendon tissue engineering
    • Acts as a good scaffold because it degrades relatively slowly and scaffold shouldn’t degrade faster than the rate of new tissue formation [3]. It is also very biocompatible.
  • Neural tissue engineering
    • Has been proven to aid in nerve regeneration

Spider silk also has desirable properties for use in drug delivery and its high tensile strength and excellent biocompatibility make it a good choice for a suture [7].

Why not just use spiders?

  • The spiders are very territorial, so are difficult to keep [3]
  • The silk needs to be made in large quantities to be useful

Ethical concerns

As much as I see a great potential in the use of spider silk to improve healthcare, I understand that there are ethical concerns surrounding the use of transgenic animals. The procedures the animals go through are invasive (e.g. superovulation and taking tissue samples). It is also felt by some people that altering animals in this way is disrupting the natural order of the universe. Ultimately, it is important we take into account the welfare of animals when doing this type of research.

References

[1] T. Miller, “The Spectacular Spider Goat – Goat Journal,” Goat Journal, Aug. 12, 2021. https://goatjournal.iamcountryside.com/kids-corner/the-spectacular-spider-goats/ (accessed Mar. 23, 2025).

[2] “Selective Precipitation — Overview & Examples,” expii. https://www.expii.com/t/selective-precipitation-overview-examples-8536 (accessed Mar. 24, 2025).

[3] F. Bergmann, S. Stadlmayr, F. Millesi, M. Zeitlinger, A. Naghilou, and C. Radtke, “The properties of native Trichonephila dragline silk and its biomedical applications,” Biomaterials Advances, vol. 140, p. 213089, Sep. 2022, doi: https://doi.org/10.1016/j.bioadv.2022.213089.

[4] Merriam-Webster, “Definition of BIOCOMPATIBILITY,” Merriam-webster.com, 2017. https://www.merriam-webster.com/dictionary/biocompatibility (accessed Mar. 23, 2025).

[5] M. F. Sigot-Luizard and R. Warocquier-Clerout, “In Vitro Cytocompatibility Tests,” Test Procedures for the Blood Compatibility of Biomaterials, pp. 569–594, 1993, doi: https://doi.org/10.1007/978-94-011-1640-4_48.

[6] Bronwyn Thompson, “Bionic silkworms with spider genes spin fibers 6x tougher than Kevlar,” New Atlas, Sep. 21, 2023. https://newatlas.com/materials/silkworms-spider-genes-spin-fibers/ (accessed Mar. 23, 2025).

[7] B. Bakhshandeh, S. S. Nateghi, M. M. Gazani, Z. Dehghani, and F. Mohammadzadeh, “A review on advances in the applications of spider silk in biomedical issues,” International Journal of Biological Macromolecules, vol. 192, pp. 258–271, Dec. 2021, doi: https://doi.org/10.1016/j.ijbiomac.2021.09.201.

The Controversial Future of Inter-Species Chimeras

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Before the stem cells lecture, I was not aware of inter-species chimeras. During the lecture it was proposed that they could be the solution to organ shortages. I found this an interesting proposition, and imagined a world where no one dies waiting for an organ. However, the thought growing of organs in another animal made me uneasy, and since learning about it, I’ve been wondering whether I would accept one if I needed it.

So what are they?

An inter-species chimera is an organism containing cells from two or more genetically distinct species. After further research I found that pigs are the most promising candidates for this research due to similarities in organ size.

Process of their creation for organ transplantation:

Video: me

As I learned more, I gained an appreciation for the innovation of genetic engineering. In theory, inter-species chimeras could be used to provide unlimited transplantable organs, but at what cost?

Image: mouse-human chimeric embryo with human cells labelled with green fluorescent protein by Jian Feng [1]

How far can we really go?

Despite promise with recent experiments forming tissues, inter-species chimeras face massive challenges. The high risk of organ rejection reminded me how fragile biology is and how even innovations struggle to overcome nature.

There are also numerous ethical concerns. How does this technology challenge our perspectives on humanity and morality? I thought about how others would react to the process of their creation. Recognising the controversy of this topic, I surveyed the public to gauge their opinions.

“The most egregious abuses of medical research” – George W. Bush describing human–animal chimeras [2]

The results were evenly split, reflecting the diverse range of beliefs that should be considered.

Reasons ranged from fear of the unknown to ethical concerns. Many of those in favour argued saving lives justifies their use. Chimera creation involves genetic modification and experiments that could cause pain or distress to animals. Unlike human, animals cannot give consent. Is it ethical to use them for experiments solely for human benefit? Before their use, key concerns must be addressed first.

“The more you can show that it stands to produce something that will actually save lives … it shifts the scale of risk-benefit assessment in favour of pursuing research and away from those concerns that are more philosophical” – Vardit Ravitsky, a bioethicist [3]

To alleviate concerns, regulatory measures could be enhanced, implementing welfare assessments. Involving the public in decision-making would also help align advancements with societal values. However, even different countries have different opinions, and religious and cultural perspectives are also highly diverse.

It is clear that this topic needs to be approached sensitively, considering both individual beliefs and societal values.

What other choice do we have?

The demand for organ transplants is not decreasing. So, what are our other options? One possible solution is in-vitro stem cells allowing for patient-specific organs. Integrating techniques such as 3D bio-printing could also help us move towards a brighter future. [4]

Image: 3D bio-printing used to synthesise tissues [5]

My final thoughts

I’m torn between saving lives and animal welfare. After more research, the complexity of the issues is only more apparent. The number of articles expressing concerns has greatly impacted my perspective, highlighting the key considerations needed before implementation.

I believe we should innovate, but within boundaries. Should we set clear limits on inter-species organ growth, and if so, what should those be? Whilst some have argued the potential to save lives outweighs concerns, I don’t think it is that clear cut. The question isn’t just whether we can create inter-species chimeras, but whether we should.

References:
[1] https://www.the-scientist.com/chimera-research-opens-new-doors-to-understanding-and-treating-disease-71254
[2] https://www.pnas.org/doi/10.1073/pnas.1615787113
[3] https://www.washingtonpost.com/news/speaking-of-science/wp/2017/01/26/scientists-create-a-part-human-part-pig-embryo-raising-the-possibility-of-interspecies-organ-transplants/
[4] https://www.sir.advancedleadership.harvard.edu/articles/engineering-high-tech-solutions-organ-shortage#:~:text=Joseph%20Vacanti%2C%20Professor%20of%20Surgery,an%20interdisciplinary%20field%20that%20applies
[5]https://blogs.manchester.ac.uk/mioir/2023/06/09/can-3d-bioprinting-cure-organ-trafficking/