It’s perhaps not the catchiest name, but it is one worth remembering: CRISPR-Cas9. Only discovered in 2012, this powerful genome editing technique has rapidly become a widespread and indispensable tool for research, and today is used in labs across the world to study human development, fertility and disease. The technology is progressing at such speed, it won’t be long until it’s a household name.
While it isn’t the first tool that has allowed us to remove, replace or add to parts of the DNA sequence, CRISPR-Cas9 is by far the most efficient, inexpensive and easiest to use, enabling precise genetic manipulation in practically any living cell.
Potentially we could use it to treat genetic disease by making changes to living, non-reproductive cells in a patient (somatic editing), or even to prevent disease through editing problematic genes in sperm, egg cells or fertilised embryos (germline editing). In the latter case, changes made to the genome would be passed on to subsequent generations.
Understandably, this has made CRISPR-Cas9 the focus of much attention – and an area of extensive ethical and philosophical debate. On the one hand, it offers the potential to rid the world of some of the most devastating inherited diseases. But some are concerned that this would mark the beginning of a slippery slope towards “designer babies” – engineered to have particular attributes such a particular eye colour or physical prowess.
To help allay these concerns, the US National Academy of Sciences was tasked with reviewing human genome editing and coming up with a set of recommendations for how it should be governed. Its report – published this week – outlines a broad set of principles that the world should follow to ensure that genome editing progresses in a cautious and sensible way, taking into account the views and opinions of the public and allowing for a variety of cultural interpretations.
Wellcome was one of the sponsors of the report. As the world’s second highest-spending charitable foundation, we’re proud to support the development of cutting-edge science. But we also believe that new technologies should be used responsibly and in a way that is culturally sensitive.
We think that the principle-led approach is the right one: it’s a model that has worked well in other areas of science, and where regulations vary from country to country, it will allow for a degree of global harmonisation, permitting some variation in practice while promoting transparency and knowledge sharing – all key to maintaining public confidence.
Importantly, the report doesn’t – as some had feared – recommend an outright ban or ‘moratorium’ on germline editing, but instead suggests that clinical trials of such treatments could, with stringent oversight, be permitted in the future for serious conditions. The principles strike the right balance: they’ll allow science to progress cautiously, but will ensure that genome editing is applied only when really needed to prevent serious conditions, such like Huntington’s disease.
But perhaps one of the strongest messages to come out of the report is the importance of public engagement – of involving all sectors of society in a meaningful discussion about the impact of genome editing technologies to help inform policy making.
This is an argument that has been made before, and one which draws upon past experience: public engagement on the topic of mitochondrial donation was key to securing understanding of and support in the UK, which in turn allowed the treatment to be regulated progressively. In time, this will change lives. We need to see the same action with genome editing.
We’ve already made a start. Wellcome is funding research with patient groups to discover what language, concepts and illustrations are most effective for communicating complex genome editing technologies, including CRISPR-Cas9.
The next step is to take these methods and see how they apply to the wider public. We’ve also commissioned a review of other reproductive technologies and their engagement strategies to help us learn from their successes and their failures.
There’s also a lot more research needed on the science itself – to understand potential unintentional changes to the genome that might result from using techniques such as CRISPR-Cas9, and to improve our understanding of how these genes link to disease. We must be able to prove that genome editing can fulfil the strict criteria laid out by the NAS: that there’s a valid case for using these technologies to combat serious disease.
So what happens next? Well, once we understand the best way to engage people on the topic of genome editing, we need to start talking with them. Ultimately it’s for society to decide how we use these powerful new tools, but we need to make sure that people have all the information required to make an informed decision.
