Human genome-editing research should proceed, say leading UK science bodies

The Academy of Medical Sciences and other leading UK research organisations have today issued an initial joint statement in support of the continued use of CRISPR-Cas9 and other genome editing techniques in preclinical research.

Genome editing is a powerful research tool that allows sections of DNA in the genome to be precisely removed or replaced using “molecular scissors”. Among these, the CRISPR-Cas9 system is a novel and highly efficient version which brought more simplicity to the process.

Our understanding of CRISPR-Cas9, though, is still at an early stage, and any potential treatments based on this technique are a long way from the clinic.

The Academy, with the other signatories, will continue to fund and support research of this kind, as well as studies that further progress and refine these technologies.

The group also calls for widespread discussion among scientists, ethicists and the wider public about how these emerging techniques may in future be applied clinically, to treat or prevent serious genetic disease.

Professor Sir John Tooke PMedSci said: "Genome editing is an evolving technology that has been used in the lab for many years to increase our understanding of disease mechanisms, and may provide the potential means to develop new medical treatments in the future.

"We believe such therapeutic use should be explored but several scientific and ethical questions remain to be answered and much more evidence will be needed before an informed decision can be made about a particular application.

"In the meantime, it is important that an open debate takes place that involves not just researchers, but ethicists, healthcare professionals, regulators, relevant patients and their families, and the wider public."

Applications of CRISPR-Cas9 may involve editing the DNA in non-reproductive cells, removing them from the body, editing them to correct a harmful mutation and then put back into the patient. There is already some work in progress to do just this in diseases including HIV, sickle-cell disease and haemophilia.

Other clinical uses of the technique might include editing the DNA of human eggs and sperm, or early embryos (known as germ-line editing). But in this case, any changes made would be passed on to future generations, which is unlikely to be allowed under current law.

Aisling Burnand, Chief Executive of the AMRC, said: “Although currently at an early stage, this technology has the potential, in future decades, to help people with devastating diseases. Patients and their families will clearly want to be involved in discussions to consider the feasibility, safety and desirability of clinical applications as the research matures.”

Professor Melanie Welham, BBSRC’s Science Director, said: “Genome editing in a preclinical research setting can widen our understanding of basic biology, which is vital to underpin advances in prolonging healthy life and disease management.”

Dr Rob Buckle, Director of Science Programmes at the MRC, said: “The UK’s place as a global leader for research is underpinned by a strong regulatory framework. As genome editing technologies evolve it’s vital that the regulatory framework remains robust and adapts so that the full potential of genome editing can be realised in a scientifically, ethical and legally rigorous way.”

Katherine Littler, Senior Policy Advisor at the Wellcome Trust, said: “As with any emerging technology, the potential for genome editing to be applied as a therapeutic tool in future deserves careful consideration. It’s essential that we start these discussions early, by engaging in an open and inclusive debate involving scientists, ethicists, doctors, regulators, patients and their families, and the wider public.”

Today’s statement was signed by the Academy of Medical Science, the Association of Medical Research Charities, Biotechnology and Biological Sciences Research Council, Medical Research Council and the Wellcome Trust.

For more information, please visit our policy project page on genome editing.

For further information, please contact:

Giorgio De Faveri at the Academy of Medical Sciences
T: 0203 1762 180 M: 07885903528
E: giorgio.defaveri@acmedsci.org.uk

Notes for Editors

1. The Association of Medical Research Charities (AMRC) is the national membership organisation of leading medical and health research charities. We help our members to meet their charitable objects and to influence the policy environment, so that the UK has the best possible climate for medical research and the translation of that research into life saving cures. We represent over 120 member charities who collectively invest over £1.3bn each year in research.
2. The Academy of Medical Sciences is the independent body in the UK representing the diversity of medical science. Our mission is to promote medical science and its translation into benefits for society. The Academy’s elected Fellows are the United Kingdom’s leading medical scientists from hospitals, academia, industry and the public service. We work with them to promote excellence, influence policy to improve health and wealth, nurture the next generation of medical researchers, link academia, industry and the NHS, seize international opportunities and encourage dialogue about the medical sciences. www.acmedsci.ac.uk
3. Medical Research Council. The heart of our mission is to improve human health through world-class medical research. To achieve this, we support research across the biomedical spectrum, from fundamental lab-based science to clinical trials, and in all major disease areas. We work closely with the NHS and the UK Health Departments to deliver our mission, and give a high priority to research that is likely to make a real difference to clinical practice and the health of the population.
4. The Wellcome Trust is a global charitable foundation dedicated to improving health. We provide more than £700 million a year to support bright minds in science, the humanities and the social sciences, as well as education, public engagement and the application of research to medicine.
5. BBSRC invests in world-class bioscience research and training on behalf of the UK public. Our aim is to further scientific knowledge, to promote economic growth, wealth and job creation and to improve quality of life in the UK and beyond.
   Funded by Government, and with an annual budget of around £484M (2013-2014), we support research and training in universities and strategically funded institutes. BBSRC research and the people we fund are helping society to meet major challenges, including food security, green energy and healthier, longer lives. Our investments underpin important UK economic sectors, such as farming, food, industrial biotechnology and pharmaceuticals. For more information about BBSRC, our science and our impact see: www.bbsrc.ac.uk. For more information about BBSRC strategically funded institutes see: www.bbsrc.ac.uk/institutes.