Genetic modification and genome editing has revolutionised biomedical research in recent decades, but the discovery and application of new technologies has led to a further step change in research in the last 2-3 years.
This has raised ethical questions, particularly around the possible application of the techniques to human germ lines or embryos, and has even led for a call for a moratorium on such research. Yesterday, five of the largest biomedical organisations and funders in the UK released a joint statement outlining their support for genomic editing research, including that involving human embryos, as long as it is legal, justified and subject to rigorous scientific and ethical review.
They also make a call for inclusive international debate on this issue. In the coming years these techniques will play an increasingly important role in our research and development of new therapies, in the pharmaceutical industry as well as in academia. It is vital that the scientific community across the life sciences sector, policymakers, patients, and the public are brought together to discuss the possibilities and ethical challenges of these techniques, to ensure appropriate and ethical progress of health research. We therefore welcome the joint statement released yesterday, and support the call for widespread debate on this issue.
Genome editing is not new - it has been used in bioscience research for several decades, allowing scientists to explore the role of different genes in health and disease, and opening the possibility of gene therapy as a therapeutic option in some diseases. However, the process was slow and laborious, and it could take many months to create a novel cell line or animal model with an edited genome. The discovery of the CRISPR-Cas 9 system in the early 2000s, and its more recent application to genome editing in mammalian cells, has revolutionised this. The CRISPR-Cas 9 system is where a molecular pathway which occurs naturally in bacteria to deal with invading viruses can be exploited by scientists to rapidly and precisely modify the genome of other cells, such as removing or replacing a gene of interest,
This technique has been rapidly taken up by scientists, both in academia and in industry, due to its great potential for helping us understand more about health and disease. For example, it can allow the rapid creation of new cell lines or animal models with a genetic mutation which causes a particular disease in humans, allowing the processes underlying the disease to be explored in that model. It also has the potential, although much further in the future, to be used in a therapeutic setting. In recent cases, scientists have used the technique to modify immune cells to target and destroy cancer cells. In the future, such cells could be administered to humans as a potential cancer treatment. These possibilities mean that research into and using such techniques should be supported for the great and exciting potential they have to bring step changes in therapeutic options for patients.
The most controversial application of such genome editing technologies though is its possible use to modify human germline (reproductive) cells, or human embryos. This raises a number of ethical concerns, including around the possibility of creating 'designer babies' and determining the genetic make up of future generations. Such work though may lead to the ability to 'correct' a genetic mutation in an embryo which would otherwise lead to debilitating disease in the person. This has led vastly divergent opinions in the bioscience sector on the application of the technique. Some scientists, including several closely involved in the development of the technique, have called for a moratorium on such research. In contrast, a research group in China inflamed the controversy when they published their work editing the genome of human embryos to modify the genetic mutation responsible for a potentially fatal blood disorder earlier this year. It is important to note that it would currently be illegal in the UK to apply this technique clinically, and is unlikely to be permitted in other European countries; such genome editing techniques would only be legal in the UK on human embryos up to 14 days old, and as regulated by the Human Fertilisation and Embryology Act 2008, and only if scientifically and ethically justified.
In their joint statement, the five biomedical organisations call for a measured approach, allowing research to continue within a tightly regulated framework, and the initiation of global debate on the issue. We agree that it is vital that ethical debate is facilitated; both the therapeutic possibilities and great ethical challenges of these techniques are too great to be ignored. Engagement must involve scientists from across the life sciences sector including industry, ethicists, policymakers, research funders, and regulators, as well as patients and the public who will ultimately be those who will be affected by these techniques. We look forward to the progress in this area, and anticipate our industry contributing actively to the dialogue.