Inside researchers’ amazing — and terrifying — gene editing discovery

news.com.au - WELCOME to the brave new world of gene editing.

An international team of scientists in the United States have safely repaired a gene mutation that causes a heritable heart defect in human embryos — sparking debate about the new frontier of genetic engineering.

The first-of-its-kind research, which was spearheaded by the Oregon Health and Science University and published last week in the journal Nature, could one day help families affected by inherited diseases.

“I, for one, believe, and this paper supports, the view that ultimately, gene editing of human embryos can be made safe. Then the question truly becomes: If we can do it, should we do it?” said Dr. George Daley, the dean of Harvard Medical School.

One major fear is that this kind human embryo modification could give rise to “designer babies,” allowing parents to pay for desirable traits they want in their kids. “I think gene editing can be used to help people who are sick,” Marcy Darnovsky, director of the Center for Genetics and Society said.

“But the idea of using it on the front end to engineer a future generation — we need to draw a bright line there.”

She insisted that current embryo-screening technology, done routinely at in-vitro fertilisation clinics across America, already helps parents avoid passing on genetic diseases to their kids.

“If you’re worried about passing on some inherited disease, you can already do that without mucking around with your child’s genes,” she said.

David King, of the Human Genetics Alert, a UK-based organisation, said governments need to “wake up and pass an immediate global ban on creating cloned or GM [genetically modified] babies before it is too late.”

“If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first GM baby,” he said.

But Shoukhrat Mitalipov, an embryologist at OHSU who led the gene-editing experiment, said the research was about “correcting” genes that cause diseases, not altering them.

“Really, we didn’t edit anything. Neither did we modify anything,” Mitalipov said. “Our program is toward correcting mutant genes.”

The researchers used a gene-editing tool called CRISPR-Cas9 — which acts like a pair of “molecular scissors” — to target a mutation that causes hypertrophic cardiomyopathy, a disease that weakens the heart and has led to the sudden deaths of many apparently healthy young athletes.

They then injected sperm from a donor with the heart disease, which affects 1 in 500 people worldwide, into eggs from 12 healthy patients, along with the genetic scissors to snip out the mutated gene. Scientists were surprised to discover the embryos then repaired themselves, taking a healthy copy of the gene from the egg as its cells began to multiply.

“The embryos are really looking for the blueprint,” Mitalipov said. “We’re finding embryos will repair themselves if you have another healthy copy.”

All told, the experiment was successful in 42 of the 58 embryos used, about 72 per cent of the time.

Mitalipov now hopes the strategy could one day be used to prevent a slew of heritable diseases caused by gene mutations, which include Huntington’s disease and cystic fibrosis. (ontinueReading