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Media reports are stating that a group of researchers in the US have successfully gene edited human embryos.
Dr Simon Waddington, Reader in Gene Transfer Technology, UCL, said:
“It’s very difficult to be able to comment on any specifics or the robustness of the science because there is no scientific paper.
“In general terms, patients with genetic diseases often have different genetic mutations. So if it was the case that CRISPR could be used to correct certain diseases, we’d need to be able to design the molecular work individually for each patient – we couldn’t necessarily make a generic treatment for a disease; it would need to be a highly personalised medicine.
“With IVF you can currently screen embryos for some diseases – pre-implantation genetic screening. We can already do PGS for a number of single gene diseases – this is much less technically challenging than gene editing, so where possible it would remain the first option.
“In general, we still don’t know enough about the functions of genes involved in lots of diseases. If it was possible to gene edit lots of genes we wouldn’t know without more research whether this would result in more benefit than potential harm.
“We are currently still at the foothills of understanding the full functions of lots of genes. If what has been reported in the media is backed up by a paper published in a peer-reviewed journal, it would be a valuable increment but we still have a long way to go.”
Dr Ewan Birney, Associate Director, European Bioinformatics Institute (EMBL-EBI), said:
“This reported piece of work is one in a line of examples reportedly showing that CRISPR based gene editing can work in cells in early stage human embryos done by a US laboratory, something which has already been shown from previous work in China. In the UK, scientists applied for and were granted a license to do this technology from the HFEA, only for research use.
“As the paper is not available it is hard to assess the technical details, but from what I can read in the media reports, it is very similar to the published work from China.
“CRISPR/cas9 gene editing can be used in a variety of ways in many different species. The major use at the moment is in research to make very precise changes in cell lines and experimental animals to study living processes. The ease of use of CRISPR makes the long-standing discussions about the appropriate ethical use of genetic changes far more relevant, both in the setting of human health and in the setting of plant and animal breeding. It is currently hard to imagine scenarios where CRISPR based gene editing is a better option than the established pre-implementation embryo testing for severe disease in humans, and we are just in the infancy of our understanding more generally how the genome relates to physical disease and attributes.
“In the UK, research and development is tightly controlled by the HFEA, a statutory body with a broad representation from UK society. The HFEA is widely regarded as one of best regulatory frameworks where cutting edge research can develop but also society has the chance to debate and control what this technology is used for. It is clear we will need an on-going dialogue across society to make best use of this technology.”
Prof. Shirley Hodgson, Professor of Cancer Genetics, St George’s, University of London, said:
“This is clearly an interesting media report but it is very difficult to be able to judge the merits of the reported work since it has not as yet been published, and there are few details of the actual results of the research available in the reports.
“The CrispR technique is known to be able to replace a faulty gene with a normal one, but so far from previous research done in China it has been a technique with significant errors in human embryos with regard to accuracy, resulting in ‘off-target’ mutations in other genes, and mosaicism in the developing embryo, since usually the ‘repair’ has only occurred in a proportion of embryonic cells.
“The media reports today suggest that in the experiments cited, these errors have been minimised, but clearly as well as needing confirmation in a published scientific paper this requires substantial further experimentation to confirm that this is actually the case. The potential extension of such research is that genetic defects in embryos can be ‘corrected’, thus removing a genetic condition from that embryo at the single cell stage. Clearly however there are many difficulties with using such techniques clinically as the CrispR manipulations would have to be done at the first cell stage of the development of the embryo, meaning that it would involve IVF and prior diagnosis that the single cell had the unwanted mutation; if this is being done, why not fertilise several eggs and chose the embryos that naturally are unaffected, which would be a one in two chance with an autosomal dominant condition for example, and avoid the need for this complex and fallable technique?
“Further ethical questions arise in discussing such techniques, such as which genetic qualities or faults are desirable and which undesirable, and these would be of concern should genetic manipulations of this kind become commonplace in future. This highlights the need for careful regulation of such technology, which we do have in the UK.”
Prof. Frances Flinter, Professor of Clinical Genetics, Guy’s & St Thomas’ NHS Foundation Trust, said:
“It is impossible to comment on a reported scientific development that has not been peer-reviewed and published in a reputable scientific journal. While it might be tempting to consider a technology that potentially offers the prospect of curing serious genetic diseases at such an early stage of development, there are major risks, both of technical failure and unanticipated adverse consequences, which could affect generations to come. Couples who wish to avoid passing on serious inherited conditions may consider well established alternatives, such as prenatal diagnosis and pre-implantation genetic diagnosis.”
Dr Helen O’Neill, Programme Director of Reproductive Science and Women’s Health, Embryology, IVF and Reproductive Genetics Group, UCL said:
“It is difficult to comment on this as there is extremely limited information available in the media reports about this (as yet) unpublished work and it relies on one attributed quote from a researcher in a collaborating group. Unfortunately, the news about the potential ability to correct disease has been eclipsed by the fear of so-called ‘designer babies’. The technology to support research into correcting diseases is readily available and is largely limited only by legislative barriers.”
Declared interests
Dr Simon Waddington: “No interests to declare.”
Dr Ewan Birney: “I am a paid consultant to Oxford Nanopore Technology, a DNA sequencing company and non-executive Director of Genomics England.”
Prof. Frances Flinter: “I have no conflicts of interest.”
Prof. Shirley Hodgson: “I have no conflicts of interest.”
Dr Helen O’Neill: “No conflicts/nothing to disclose.”
None others received.