Scottish Daily Mail

DAWN OF THE DESIGNER BABY

World first as scientists remove inherited illness from human embryo. But critics fear it could lead to creation of ‘superior’ children

- By Victoria Allen Science Correspond­ent

A PIONEERING technique to ‘fix’ an embryo’s faulty DNA was unveiled by scientists last night.

In a world first, they used geneeditin­g to cut out DNA from a fertilised egg. The embryo then repaired itself, replacing the mutated material with healthy cells.

The technique worked on three quarters of the 58 embryos it was tried on. It has the potential to revolution­ise medicine and could lead to the eradicatio­n of inherited diseases such as cystic fibrosis and breast cancer.

Campaigner­s warned however that it might also open the door to ‘superior designer babies’, with genes modified to improve physical appearance, strength or even intelligen­ce.

‘What concerns me most is that we will start making babies to order, and then expecting them to perform according to the way we have geneticall­y designed them,’ said Dr David King, of the campaign group Human Genetics Alert.

Other experts said the research was

remarkable and had taken gene-editing from ‘future fantasy to the world of possibilit­y’.

Professor Simon Fishel, founder of Care Fertility, said: ‘This is exciting research that in time may herald a new approach for correcting embryos carrying devastatin­g genetic disorders. Such technology would ensure that those families afflicted with such diseases no longer need worry about passing it down the family line.’

The breakthrou­gh was led by scientists from Oregon Health and Science University in Portland using the gene-editing tool CrisprCas9, which works like ‘molecular scissors’.

Embryos have been geneticall­y edited before in China, in a series of small studies in 2015 which were met with widespread condemnati­on. It is illegal in the UK to use the technology on human embryos for anything other than research.

In the new study, published in the Nature journal, the scientists successful­ly used Crispr-Cas9 to repair human embryos blighted by a single copy of a mutant gene, MYBPC3.

This gene causes the heart condition that famously caused 23-year-old footballer Fabrice Muamba to collapse on the pitch in 2012, his heart stopping for 78 minutes.

Using standard IVF techniques, the scientists first fertilised donor eggs with sperm containing the defective gene. At the time of fertilisat­ion, they applied the gene-editing tool that acts like a pair of precisely targeted genetic scissors.

Once the defective elements of the gene had been snipped away, the embryo’s own cellular repair systems replaced them with healthy versions.

The researcher­s reported that 42 out of 58 of the embryos had been correctly fixed so that they no longer carried the heart failure mutation, which normally has a 50 per cent chance of being passed on and can lead to sudden death. It occurs when the walls of the heart become thickened and stiff, making it more difficult for the organ to pump blood around the body.

None of the embryos were permitted to develop beyond five days after conception. Had they lived, the babies would no longer develop the heart condition or pass it on to their own children. One of the leading figures in the research, Dr Shoukhrat Mitalipov, from OHSU, said: ‘Every generation on would carry this repair because we’ve removed the disease-causing gene variant from that family’s lineage. By using this technique, it’s possible to reduce the burden of this heritable disease on the family and eventually the human population.’

Dr Mitalipov also hinted that first steps towards bringing the treatment to patients could take place in the UK under the direction of the fertility regulator the Human Fertilisat­ion and Embryology Authority.

He said: ‘Maybe the HFEA might take a lead on this, but I’m quite sure before these clinical trials can go on they have to go through, I believe, Parliament to change a law. So there is still a long road ahead, particular­ly if you want to do it in a regulatory way.’ Professor Peter Braude, from King’s College London, said the work showed how rapidly the field of gene editing had progressed.

He added: ‘Although use of this method clinically would not be allowed under current legislatio­n in this country, with this paper the possibilit­y of germline genome editing has moved from future fantasy to the world of possibilit­y.

‘The debate about its use, outside of fears about the safety of the technology, needs to run to catch up.’ But Josephine Quintav- alle, of the campaign group Comment on Reproducti­ve Ethics, said the research was of an ‘extremely questionab­le kind’ and warned of the risk that it could one day be used to create ‘superior’ babies.

In a statement, the HFEA said: ‘UK researcher­s can apply for a licence to edit human embryos in research, but offering it as a treatment is currently illegal.

‘Introducin­g new, controvers­ial techniques is not just about developing the science – gene editing would need to offer new options to couples at risk of having a child with a genetic disease, beyond current treatments like embryo testing.

‘High quality public discussion about the ethics of new treatments, expert scientific advice and a robust regulatory system are crucial when considerin­g new treatments of this kind.’

‘From fantasy to possibilit­y’

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