Kathy Niakan, a researcher at The Francis Crick Institute in London, applied to the Human Fertilisation and Embryology Authority (HFEA), asking to lift restrictions on human gene editing.
If HFEA grants approval, the Crick’s work will be for research only and “will not have a clinical application,” the scientists said. The embryos will not be implanted to progress into a pregnancy.
The researchers said results will help them understand how a healthy human embryo develops, and “this knowledge may improve embryo development after in vitro fertilisation (IVF) and might provide better clinical treatments for infertility.” The work also has “tremendous potential for stem cell research, which will have benefits and advances in many different fields of medicine,” they added.
CRISPR
Niakan said the institute wants to use newly-developed CRIPSR-Cas9 technology to make specific changes to the genome. “By applying more precise and efficient methods in our research we hope to require fewer embryos and be more successful than the other methods currently used.
"Importantly, in line with HFEA regulations, any donated embryos would be used for research purposes only. These embryos would be donated by informed consent and surplus to IVF treatment."
The CRISPR-Cas9 technique allows more precise editing than previous methods, including “knocking-in” genes.
Expert reaction
Under UK regulations, the work proposed by the Crick – if approved – would be done in a lab with surplus IVF embryos which would otherwise be destroyed.
“In the proposed study, the first if its kind in the UK, the activity of specific human genes will be altered using CRISPR/Cas9 genome editing tools,”said Bruce Whitelaw, Professor of Animal Biotechnology at the University of Edinburgh. “Similar studies have already been done in mice. But mice are not humans, and to have a realistic chance of having more, healthy IVF pregnancy outcomes, researchers must do human embryo research.
“The recent Hinxton Group consensus statement highlighted that genome editing technology has tremendous value as a tool to address fundamental questions in human biology. The current application to the HEFA illustrates that British science aims to embrace the opportunity to use genome editing technology to enhance medicine.”
Most national regulators currently ban gene editing in humans, but scientists from Sun Yat-sen University in China claimed in Protein & Cell in May this year to have edited the genomes of non-viable human embryos.
‘Ethical questions’
In August, a joint statement from the Wellcome Trust, Medical Research Council, Association of Medical Research Charities, and other groups said they support the current use of genome editing in preclinical biomedical research, but extending its scope requires careful thought.
“Within the UK, this research may involve the use of somatic (non-reproductive) or germ cells, including human embryos up to 14 days old – within the confines of the HFE Act 2008 – where appropriately justified and supported by rigorous scientific and ethical review.
“We believe that genome editing technologies may hold significant potential for clinical application in the future; and we would be open to supporting the development of new therapeutic approaches should the evidence from research advance sufficiently to justify their use.”
The groups said they recognise future potential to use genome editing in a clinical context using human embryos, although “this is prohibited by law in the UK and unlikely to be permissible in other European jurisdictions at present.”
Ethical and regulatory questions surrounding this research should be explored by scientific experts and the public before decisions are made about clinical applications, they said.