Human gene editing work should continue say major UK research funders

A group of leading UK research organisations has issued a joint statement in support of the continued use of genome editing technologies such as CRISPR in preclinical research. This includes the use of the technology for research purposes in human eggs, sperm and early embryos, in cases where this is fully justified both scientifically and ethically, and is within the confines of the law.

The group, including the Wellcome Trust, the Medical Research Council and the Association of Medical Research Charities, said it will continue to fund and support research of this kind, but called for a discussion among scientists, ethicists and the wider public about how genome editing should be used in future to modify human reproductive cells and early embryos, in order to treat or prevent serious genetic diseases.

Earlier this year Chinese scientists reported using gene editing to alter the genome of human embryos, leading to an international call for a moratorium on the use of the technique. The U.S. National Academy of Sciences subsequently launched an initiative to agree global standards and guidelines for the use of the technology.

Genome editing allows sections of DNA in the genome to be precisely removed or replaced using ‘molecular scissors’. The concept is not new, but rapid developments in the technology – namely the emergence of the CRISPR system developed by Crispr Therapeutics AG of Basel, Switzerland – mean that targeted, highly efficient editing of DNA in cells has become relatively simple.

While potential treatments based on this technique are many years away, there is already work in progress. The first clinical applications are likely to involve editing the DNA in somatic (non-reproductive) cells, removing them from a patient and editing them in a lab to correct a harmful mutation, before putting them back into the body. Research aiming to do this in diseases including HIV, sickle-cell disease, haemophilia and cancer is underway.

Genome editing could be used clinically in the future to edit the DNA of human eggs and sperm or early embryos. The difference here is that any changes made would be passed on to future generations. Such research is prohibited under UK and European law.

However, the UK Human Fertilisation and Embryology Act does permit the use of 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 statement says that this type of research should to be allowed to continue. Genome editing is an evolving technology that has been used in the lab for many years to increase understanding of disease mechanisms, and may provide the potential means to develop new medical treatments in the future, said John Tooke President of the Academy of Medical Sciences. “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,” Tooke said.

As with any emerging technology, the potential for genome editing to be applied as a therapeutic tool in future deserves careful consideration, said Katherine Littler, Senior Policy Advisor at the Wellcome Trust. “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,” she said.