Genome Editing Techniques: The Science and the Ethics

Genome Editing Techniques

A group of UK research organisations, including the Wellcome Trust, have confirmed their support for genome editing techniques in preclinical human research, including in human reproductive cells and early embryos.

Genome editing techniques such as CRISPR-Cas9, TALENs and zinc fingers, sometimes described as ‘molecular scissors’, allow targeted and efficient editing through precise removal and/or replacement of sections of genomic DNA. This is a powerful research technique, helping researchers to understand the impact of changes in the genome in human reproductive cells and early embryos.

“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,” says Professor Melanie Welham, the science director of the Biotechnology and Biological Sciences Research Council (BBSRC).

The techniques also have potential for clinical application in the future, for example, by removing cells from a patient, editing them in a lab to correct a harmful mutation, and returning them to the body. Research is under way in HIV infection, sickle-cell disease, haemophilia and cancer.

“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,” says Professor Sir John Tooke PMedSci, President of the Academy of Medical Science (AMS).

Further ahead, the techniques could be used to edit the germline in human reproductive cells and early embryos, to treat or prevent serious genetic disease. These changes would be passed on to future generations. While this approach is currently prohibited under UK law and is unlikely to be allowed in any European jurisdiction at present, the UK Human Fertilisation and Embryology Act does permit the use of new technologies such as gene editing for non-clinical research purposes in germ cells, including human embryos up to 14 days old, where this is appropriately justified and supported by rigorous scientific and ethical review.

“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,” says Dr Rob Buckle, director of Science Programmes at the Medical Research Council (MRC).

The AMS, the Association of Medical Research Charities (AMRC), the BBSRC, the MRC and the Wellcome Trust, say that this type of research should be allowed to continue.

“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,” says Aisling Burnand, chief executive of the AMRC.

The organisations plan to continue to fund and support research into genome editing, but have emphasised that any research must be fully justified, both scientifically and ethically, and within the confines of the law. They have also called for a broad and inclusive discussion about genome editing and its future implications among scientists, ethicists and the wider public.

“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,” says Katherine Littler, senior policy advisor at the Wellcome Trust.

The support of the UK research organisations is being translated into possible human disease therapy with companies such as Juno Therapeutics, Crispr Therapeutics, Editas Medicine and recently Intellia Therapeutics all developing potential disease treatments using the technology. Rainbow Seed Fund’s synthetic biology fund is an early investor in Desktop Genetics, a UK based company developing CRISPR planning software with potential applications in therapeutic development.