The Daily Telegraph

UK ‘should use gene editing to prevent diseases’

- By Rozina Sabur

BRITAIN should take the lead in the use of human embryo gene editing to eradicate inherited diseases, the scientist behind a breakthrou­gh study suggested yesterday.

Scientists in the US reached a new milestone by successful­ly altering DNA in defective embryos so they were no longer programmed to develop heart failure. It is the first time the powerful gene-editing tool Crispr-cas9 has been used to fix a mutation responsibl­e for a common inherited disease.

The highly controvers­ial technique is still at an early experiment­al stage and there is no question of any attempt being made to create babies with the genetic modificati­on, which would be illegal both in the US and the UK.

The Telegraph revealed the procedure had been carried out last week, but now a leading member of the team has 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 (HFEA).

Dr Shoukhrat Mitalipov, from Oregon Health and Science University in Portland, 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.”

US regulatory barriers to such research are so high they could be insurmount­able. Britain has blazed a trail by becoming the first country officially to sanction mitochondr­ial replacemen­t therapy (MRT), seen by some as opening the door to “designer babies”.

The treatment replaces faulty inherited DNA in the mitochondr­ia, tiny rodlike bodies in cells that supply energy, to prevent devastatin­g diseases.

However, the new research reported in the journal Nature goes much further by modifying the nuclear DNA at the heart of the cell that influences personal characteri­stics such as height, facial appearance, eye colour and intelligen­ce. Leaked informatio­n has already led to widespread speculatio­n about the study. The published paper explains how Crispr-cas9 was used to repair human embryos blighted by a single copy of a mutant gene, MYBPC3, that causes hypertroph­ic cardiomyop­athy, a type of heart failure.

But Dr David King, director of the Human Genetics Alert, which opposes all tampering with the human genome, said: “If irresponsi­ble scientists are not stopped, the world may soon be presented with a fait accompli of the first GM baby.”

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