Genome-editing research: not just a question for scientists

The Wellcome Trust is proud to support the development of cutting-edge science, but we are also aware of the importance of ensuring that new technologies are used responsibly. The CRISPR-Cas9 genome-editing process and its applications in research are an area of growing interest.

Ahead of a range of international meetings and consultations, Wellcome is today outlining its initial position on the technique. [PDF 87KB] Senior policy adviser Katherine Littler explains.

Credit: Wellcome Library, London

The CRISPR-Cas9 technique for genome editing has been in use by researchers for around three years, and as awareness of the technique has grown, potential new applications are being considered.

The technique exploits a naturally occurring phenomenon, to allow sections of an organism's DNA to be accurately and efficiently targeted, then removed or replaced using 'molecular scissors'. Researchers are now using the technique to help them better understand the function of different genes, and the effect they have on health and disease.

The ability to edit genes for research is not new, and there are various techniques already in use, but with the potential for CRISPR-Cas9 to make the process more efficient, many people are thinking about how it could be used therapeutically. One use that has attracted particular attention is the possibility that it could one day be used clinically in human embryos, to prevent serious inherited conditions.

In reality, this is a long way off, we are still in the early stages of understanding the full potential of CRISPR-Cas9 and more research needs to be done.

While there has been some good progress developing potential somatic (non-reproductive) cell therapies using this technique, it will be years before we know enough to apply it safely in reproductive cells.

But, at the Wellcome Trust we believe it is important that discussions about the application of new scientific techniques start early, and are not limited to the community of scientific researchers who are currently using them. We want to encourage wider engagement with the questions that new techniques may raise, and we feel that it is important to do this as early as possible.

We also believe that the discussions about the potential applications of genome editing should not stand in the way of basic research that makes use of the technology to inform our understanding of human biology, health and disease.

Today we are publishing our initial position on the technique. [PDF 87KB] In short we would like to make it clear that we support the development of new techniques and applications of science that could help improve human and animal health, but we want to do this in the context of wider discussions that should be had about how and where the technique should, and shouldn't, be used.

Biomedical and social science researchers, ethicists, healthcare professionals, funders, regulators, patients and families and the public should all have a voice in this discussion.

The joint position statement that we have released today, (also signed by AMRC, AMS, BBSRC, and MRC) is an initial statement to show that we want to be engaged in, and help to facilitate discussions on this topic. We fully expect this to evolve and be refined as the conversation (and the research) moves forward.

We know that CRISPR-Cas9 is already an effective tool in basic research, and in future could be used to treat disease, but we are not there yet – not scientifically, ethically or legally. As the technology evolves, we should maintain the robust regulatory framework that has already allowed the UK to become a respected global leader in the application of science in an ethical manner

We want to proactively engage with the wider questions raised by genome editing, and feel that the best way forward at this point is to encourage open discussion that includes scientists, ethicists and the public. We look forward to being part of that discussion and would welcome your thoughts.