Why Nobel Prize-winning CRISPR should be the game-changer for animals
Published on October 19, 2020
Earlier this month two pioneering researchers, Professor Emmanuelle Charpentier and Professor Jennifer Doudna, won the Nobel Prize for Chemistry for discovering and developing an innovative new research technique known as CRISPR/Cas9 – better described as ‘genetic scissors’.
The scientists ‘rewrote the code for life’ with their revolutionary gene-editing technology in 2012, and it has since swept through the scientific community, revolutionising basic research and promising ground-breaking medical treatments for cancer and inherited diseases.
Animal Free Research UK’s CEO Carla Owen and Science Manager Dr Alpesh Patel explain, CRISPR can be a game-changer for humans but also for animals – if researchers have the courage and imagination to do things differently.
The Nobel Prize is a just reward for Professor Emmanuelle Charpentier and Professor Jennifer Doudna because their CRISPR/Cas9 breakthrough made for many scientists a dream come true – a gene-editing tool that identifies specific sequences of DNA, cuts out ‘faults’ and replaces them with a ‘healthy’ sequence.
The tool is currently being developed as a treatment to modify a patients’ stem cells and replace defective genes – the goal being that a patient can have their ‘faulty’ stem cells removed, edited into a ‘normal’ condition, and then have them reimplanted.
The most common CRISPR technique, CRISPR/Cas9, is named for the Cas9 protein which is used to identify the target DNA sequence.
CRISPR/Cas9 has huge potential in the fight against genetic diseases such as sickle cell anaemia, cystic fibrosis, and Huntingdon’s disease, bringing much-needed hope to patients and families.
The technique is powered by proteins from bacteria which are programmed to detect a particular DNA sequence, often near a mutated gene. When a match is found, a complex of proteins and enzymes come together to cut out the DNA sequence and replace it with a new version.
Core to CRISPR’s success is its ability to be precise about the DNA being targeted. Other gene editing techniques are more clunky and have a greater risk of being ‘off-target’ – and they come with a host of undesirable side-effects if the DNA is inserted incorrectly, including cancer.
Undoubtedly, CRISPR is one in a range of exciting next generation research tools that enable scientists to accelerate away from traditional research as part of the increasing take up of new approach methodologies.
CRISPR and animals
Animal Free Research UK is among those who believe CRISPR is not only a game-changer for human health but it is also a powerful tool to help replace animal experiments in research.
The technology can enable scientists to modify cells extracted from patients in the lab with a high level of confidence that the correct sequence of ‘faulty’ DNA has been replaced with ‘healthy’ sequencing. This is human-relevant research at its best, delivering effective treatments for humans without animal suffering.
Yet as we join in congratulating the pioneering researchers on their Nobel Prize, we also harbour significant concerns about the use of CRISPR in animals from a scientific as well as ethical perspective.
Because as with all new research tools, advancing technologies which empower researchers to work better science can also be used to harm as well as benefit society.
Sadly, CRISPR is being used to genetically mutate animals – often multiple times – in an attempt to ‘humanise’ them by introducing human DNA into their bodies.
Indeed, many researchers are specifically using CRISPR to genetically modify animals. We’ve seen a huge increase in the number of animals genetically altered since the technique was developed in 2012 – a staggering 1.66 million in Britain alone in 2019.
Scientists can too easily fall into the trap of applying new technology to outdated animal-based research, rather than creatively exploring how they can transform their thinking in light of these new developments.
When a human gene is introduced into an animal through a technique like CRISPR, that gene may be used quite differently in the animal than in humans. For example, a study published in Science found that when a crucial human protein which controls blood sugar movement in humans, and which is naturally missing in mice, was introduced into genetically altered mice, it behaved very differently.
Rather than helping to control their blood sugar, the gene caused impaired blood sugar control in the animals. Inevitably, human genes genetically edited into animals will be affected by the many and varied physiological mechanisms that are unique to those animals. Humanised animals are not humans.
So rather than wasting precious time, money and animal lives trying to humanise animals in an attempt to treat human diseases, cutting-edge tools like CRISPR and other new approach methodologies should be used to find truly human-relevant solutions.
CRISPR can be used to modify human cells in the laboratory, in cell culture and organ-on-a-chip models to deliver data from human sources that is directly relatable to human diseases.
We urgently need to accelerate towards personalised medicine that is patient-focussed, and not rely on genetically altered animals who can never accurately replicate a human patient in all their rich complexity.
The only solution – both ethically and scientifically – is to invest in human models of disease. That’s why Animal Free Research UK is funding a pilot study using CRISPR to create human stem cell disease models for the study of vascular dementia at the University of Manchester.
And it’s why we have a new three-year project about to launch at the University of Nottingham that will develop stem cell models to understand heart disease using CRISPR.
Nobel Prize winning CRISPR can be a game-changer for animals as well as for patients – if researchers have the courage and imagination to do things a little differently.