Genome editing moves into the spotlight

Hilary Burton

3 February 2016

The PHG Foundation has submitted a response to the Nuffield Council on Bioethics consultation on genome editing. 

The technology has once again dominated the news this week as the Human Fertilisation and Embryology Authority (HFEA)  announced that it has approved a licence to use gene editing of embryos in research, subject to ethical approval. Dr Kathy Niakan of the Francis Crick Institute in London will carry out experiments to study how the embryo develops shortly after fertilisation, to inform research into the causes of infertility and miscarriage. The embryos will be donated by couples undergoing IVF, and current legal restrictions remain in place: the embryos must be destroyed before they are 14 days old, and it will be illegal to use them to achieve a pregnancy. 

Scientists have been able to carry out forms of genome editing of some description for decades, but the development of a range of related new editing technologies utilising engineered bacterial proteins, most notably the CRIS PR/Cas9 system, has pushed genome editing into the mainstream. The method is easier, cheaper and quicker to use than other methods and has effectively placed the technology into the hands of many researchers, rather than an elite few teams with the funding, expertise and persistence needed to achieve success. But what are the wider implications of making this technology more accessible? 

Regulatory oversight

We are fortunate in the UK in having a solid regulatory infrastructure for research involving human embryos, which in recent years has included consideration of a range of emerging technologies and potentially controversial applications. For example, the HFEA held public consultations and heard expert evidence in examining the creation of human-animal hybrid embryos for medical research, and the application of mitochondrial transfer techniques to allow women affected by severe mitochondrial disease to conceive unaffected genetically-related children.

While we acknowledge that it is still vitally important to continue public discussions for new applications of this kind, we also think that a challenge lies ahead in terms of managing the increased workload involved in research oversight that will result from the widespread use of a more accessible technology. We must ensure that our regulatory infrastructure is able to manage this increase in use, and that we can continue to carefully investigate the impact and consequences of genome editing. 

Scientific applications

CRISPR/Cas9 and related genome editing techniques are already revolutionising scientific research, through use in the development of new cell and animal models. Depending on the genetic modifications required, mouse models that would previously have taken 1-2 years to develop can now be created in months. The long term impact of this on medical research will be difficult to calculate, but it is likely that research in certain areas will become quicker and easier to carry out. This may lead to an increasing bottleneck in the translational infrastructure necessary to move discoveries from basic science into clinical application, making it necessary to prioritise those applications most likely to result in useful new treatments.

Medical applications

Last year, the successful treatment of a one-year old girl with acute lymphoblastic leukaemia using gene edited T cells provided a tantalising glimpse into the medical possibilities of the technique, here combined with stem cell transplantation. While research into the treatment of blood and immune disorders is promising, there are many hurdles to be overcome surrounding the safety and efficacy of these techniques. One impact of CRISPR/Cas9 is that possible medical applications are suddenly closer to reality than previously expected, meaning that we must be prepared for the extensive ethical and legal discussions that are to come, as well as managing the inevitable hype that will surround the development of new therapies.

Germline genome editing

The potential for heritable, germline genetic modification using gene editing has received the most publicity, but this area is also the most tightly regulated. The scientific community has already organised discussion meetings on this topic and we believe that the collaborative communities that are developing in response to gene editing technologies should be supported and encouraged. As far as possible it will also remain vital to include a wide range of voices in these discussions; whilst scientists are best equipped to explain the true potential and limitations of the technique, reaching a societal consensus on which research applications are and are not desirable needs broader input, from public views to legal, ethical and regulatory perspectives.

As we state in our consultation response, we think that 'Genome editing…has enormous potential to speed research and facilitate useful and effective applications which could be beneficial to human health'. We believe that an approach that encourages and supports collaboration and self-regulation is likely to be more beneficial than the adoption of specific moratoria against genome editing. While the UK has strong infrastructure to manage these issues, the community-based approach and peer-to-peer influence is more likely to have an impact on researchers in countries where there is little or no regulation or ethical review.