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US lab may have edited human embryos for first time

31 July 2017

By Charlotte Spicer

Appeared in BioNews 911

Scientists in the US may have successfully used genome editing in human embryos to correct disease  mutations, according to an exclusive report by MIT Technology Review.

The results of the study are yet to be published, but the work – led by Professor Shoukhrat Mitalipov at the Oregon Health and Science University, Portland – is believed to include the largest number of successfully modified embryos produced using CRISPR technology.

Professor Mitalipov declined to comment on the study, but stated the results are pending publication next month. However, Dr Jun Wu, a collaborator at the Salk Institute, California told MIT Technology Review, 'So far as I know this will be the first study reported in the US.'

Three published attempts of genome editing in human embryos have been carried out in China to date, the first in 2015 (see BioNews 799). Professor Shirley Hodgson, a specialist in genetics at St George's, University of London, said the 2015 study had shown CRISPR to be '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.'

However, sources claim that the US study overcomes these two hurdles. Professor Mitalipov's group are believed to have injected eggs with CRISPR at the same time as they were fertilised with donor sperm, as opposed to after. The donor sperm contained inherited disease-related mutations, though it is not known which disease genes these were. It is reported that the majority of cells in the resulting embryos were successfully edited so that the disease genes were replaced with healthy genes, with few errors.

The successfully edited embryos of 'clinical quality' were discarded after a few days. The team had no intention of implanting them.

A scientist familiar with the project told MIT Technology Review, 'It is proof of principle that it can work. They significantly reduced mosaicism. I don't think it's the start of clinical trials yet, but it does take it further than anyone has before.'

Scientists are reluctant to comment on the results until they have been confirmed in a published scientific paper, yet some caution that there is still a long way to go until it would be possible to implant any edited embryos.

'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,' said Professor Frances Flinter, a clinical geneticist at Guy's & St Thomas' NHS Foundation Trust, London, who was not involved in the study.

Besides the safety issues, the study also raises important ethical questions about how the technology could or should be used. Research involving human embryo editing is barred from federal funding in the US. Careful regulation is in place in the UK, with scientists granted permission from the Human Fertilisation and Embryology Authority (see BioNews 837) to genetically modify human embryos for research use only.

Dr Simon Waddington of University College London, who was not involved in the study, concluded, '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.'

The Telegraph | 27 July 2017
Medscape | 27 July 2017
MIT Technology Review | 26 July 2017
Science magazine | 27 July 2017
STAT News | 26 July 2017


02 October 2017 - by Sandy Starr 
What do patients and laypeople think and know about genome editing and its implications? What are the best ways for experts and others to discuss genome editing in public, so as to improve public understanding and avoid confusion? The Progress Educational Trust has set out to answer these questions, with its 'Basic Understanding of Genome Editing' project....
04 September 2017 - by Annabel Slater 
A group of scientists have challenged the landmark study which reported the first successful editing of human embryos for a genetic disease...
07 August 2017 - by Charlotte Spicer 
Scientists have published their study confirming they are the first to correct a disease-causing mutation in human embryos using genome editing...

13 March 2017 - by Dr Katie Howe 
Chinese scientists have successfully used genome editing to correct mutations in viable human embryos for the first time...
26 September 2016 - by Anneesa Amjad 
A scientist in Sweden has become the first to edit genes in healthy human embryos...
11 April 2016 - by Ayala Ochert 
A second team in China report that they have created genetically modified human embryos, in an attempt to make them resistant to HIV, using the genome-editing technique CRISPR/Cas9...
01 February 2016 - by Ayala Ochert 
The Human Fertilisation and Embryology Authority has granted the first licence to a UK researcher to edit the genomes of human embryos...
27 April 2015 - by Ayala Ochert 
Chinese scientists report the first-ever genetic modification of human embryos using the CRISPR/Cas9 gene-editing technique, confirming rumours that these highly controversial experiments were underway...

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