Crossing the Rubicon

A scientist has received international condemnation in the genetics field after claiming to have produced the world’s first gene-edited babies using CRISPR technology. In videos posted by his lab to justify the work, Jiankui He, an Associate Professor at the Southern University of Science and Technology in Shenzhen, China, says the gene-edited twin girls were born as “healthy as any other babies” (1). The gene editing was conducted after IVF, when each embryo was just a single cell, to modify the CCR5 gene to block HIV infection. The girls’ father was HIV positive and He says that the twins will be monitored over the course of the next 18 years.

“I understand my work will be controversial, but I believe families need this technology and I’m willing to take the criticism for them,” He said in one of the videos. He says he targeted HIV because of “safety”, adding that CCR5 is one of the best studied genes and many people have natural genetic variation that disables the gene to prevent HIV infection. He claims it was the “simplest gene surgery possible.” Gene editing to correct broken genes to perhaps target familial cancers or muscular dystrophy would be more complex.

CRISPR’s ability to edit genes, coupled with its cost-efficient production and high degree of fidelity, make it an attractive method of genome editing, but navigating the ethical minefield associated with its use seems to be challenging for the research community. The risk of harm to human life and its potential to cause environmental damage limit its applications. Current iterations of the technology make it possible to introduce off-target mutations (which introduce mosaicism and off-target mutations (which can be deleterious) into human DNA. Forty countries including the UK, US and China have banned or discouraged the use of genome editing.

Gaetan Burgio, Geneticist and Group Leader at Australian National University, who tweeted about a panel discussion that He was involved in at the Human Genome Editing Summit where he discussed the work, said that the method He outlined doesn’t do enough to rule out mosaicism or deletions (2). The scientific community has called for an independent peer-review of the trial to assess the validity of the claims made by He.

The global scientific community fears He’s work could hinder the future of CRISPR by exacerbating the public’s scepticism and forcing the hand of regulators, compelling them to put even stricter rulings into place about the use of the technology.

The Southern University of Science and Technology says it was not aware of the project and that He had been on paid leave since February. A variety of mixed reports are now circling in the media regarding He’s location. Some have reported he is under house arrest in China, but the Southern University of Science and Technology says that this information is not accurate. China’s Vice Minister for Science and Technology has called for a suspension of any scientific activities by those involved in He’s work.