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He Jiankui’s name is not on the list of registered delegates for the Third International Summit on Human Genome Editing, to be held next month at the Francis Crick Institute in London. Still, most of those present will think of the disgraced Chinese scientist. He will be a ghost at the feast of science.
Jiankui was responsible for one of the most controversial acts in modern scientific history, as revealed at the previous world genome-editing summit, held in Hong Kong in 2018. of Science and Technology in Shenzhen, announced that he had altered the genetic makeup of three young girls in an attempt to make them resistant to HIV. This change – made when they were still embryos – could then be passed on to future generations.
The experiment is unprecedented in modern genetics and was deemed unethical by Chinese authorities. Jiankui was subsequently jailed for three years, although his influence over next month’s science summit will still be strong, said Professor Robin Lovell-Badge, organizer of the upcoming London summit.
“We will discuss what happened to the three children whose physiology he may have altered through genome editing,” said Lovell-Badge, who also chaired the session in which Jiankui unveiled his extraordinary biological intervention. “We will also be giving presentations on the changes that have occurred in China in terms of the law and ethics related to gene editing. There have obviously been some pretty substantial changes – for the better.
“And it is important that these issues are raised. Genome editing has enormous power to benefit people, but we need to be transparent about how it is tried and tested before putting the technology into practice.”
Genome editing was transformed by Jennifer Doudna of the University of California, Berkeley, and Emmanuelle Charpentier, of the Max Planck Institute for Infection Biology in Berlin. Their research was rewarded in 2020 when the pair received the Nobel Prize in Chemistry for creating “a technology [that] has revolutionized molecular life sciences,” said the Royal Swedish Academy of Sciences, which presented the award.
The technique developed by Doudna and Charpentier is known as Crispr-Cas9 and works like molecular scissors that can cut a DNA strand at a specific spot. In this way, scientists can alter the structure of genes in plants, animals and humans, and in turn induce changes in physical traits, such as eye color, and disease risk. There is no insertion of genes from other organisms, a crucial difference from previous forms of genetic engineering.
Scientists are now looking at genome editing to develop new medical treatments, for example by making changes in people with diseases. Candidates include the inherited disease sickle cell anemia, in which a single gene defect disrupts hemoglobin production with serious consequences for patients, who suffer from anemia because their bodies are deprived of oxygen.
Removing a person’s stem cells and then genetically editing them so that they begin producing fetal hemoglobin — a process normally turned off at birth — could restore red blood cells to their bodies, scientists believe. Trials are already being carried out in several centres.
In addition, doctors and researchers are exploring ways to use genome editing to address muscular dystrophy, cancer, diabetes, some forms of hereditary blindness, and many other debilitating conditions that have defied previous attempts to cure them. At next month’s summit, hundreds of delegates will gather to hear the latest developments.
Other experts look even further into the future. One idea is to change the physiology of astronauts so they are better protected against radiation and the effects of weightlessness, invaluable for travel to Mars and beyond.
“You could also think about modifying liver enzymes to make men and women better able to rid their bodies of toxins used in chemical warfare, or making changes that would make them more resistant to biological weapons,” he added. Lovell Badge to it. “That’s the kind of human enhancement that military researchers are thinking about right now.
“You could also consider altering humans so that they can see in the infrared or ultraviolet range, as some animals can do. Such improvements would be ideal for troops fighting at night or in other hostile conditions.”
To what extent such human improvements will be tolerated by society is another matter – one that will be addressed at a separate event at the Francis Crick Institute. A public exhibition entitled Snow + Slices, will investigate which changes can be made safely in humans using genome-editing technology; which priorities should be assessed and which can be considered morally unacceptable and excluded from future research.
“Genome editing tools hold enormous potential to improve human health and the world around us, but like all new technologies, they raise ethical questions and concerns,” said Ruth Garde, curator of Cut + Paste, which opens this week. “The public doesn’t know much about these techniques at the moment. Cut + Paste will enable visitors to explore and reflect on the ethics of genome editing through a series of interactive experiences.”
The exhibition will cover all aspects of genome editing, including its use in improving crops and farm animals, although the impact on humans will be the focus, as will be the case at next month’s international summit. “Genome editing has made it easier to imagine human traits ‘improving’,” Garde added. “Cut + Paste leaves visitors wondering what “improvement” means? What is a ‘desirable’ trait? And who decides that?”
Visitors are asked to consider various applications of genome editing: to tackle diseases such as malaria by using the technology to render mosquitoes infertile; to enhance human capabilities; and to make physiological changes that are passed down from generation to generation. “Critically, we invite visitors to tell us what they think of these ideas,” said Garde.
These issues will also be the subject of keen debate at the summit. “Genome editing for sickle cell anemia has great potential, but it is also very expensive,” says Lovell-Badge. “Treatment for one person can cost a million dollars. However, the disease is most common in Africa, where people can afford the least expensive medicines. So are we in danger of creating even bigger health gaps between developed and developing countries? It is a major concern.”
The rogue activities of Jiankui, who has since said he acted “too quickly”, will add further excitement to the discussions. “Jiankui is now out of jail and running a lab in Beijing again,” Lovell-Badge said. “He says he will focus on gene therapy to treat diseases like muscular dystrophy. And that scares me, because he’s not a biologist. He knows little about the disease.”
As for the motivation for Jiankui’s past actions, the scientist has since pointed to the fact that infection with HIV can lead to people being ostracized. He wanted to get around that. “He chose to try and make changes to the CCR5 gene, which has naturally occurring variants that can protect against HIV. In this way he hoped to create genetically engineered protection,” said Lovell-Badge.
“But experiments have also shown that in about 20% of cases, these genome-editing changes can lead to substantial rearrangements of a person’s genome, which is very, very dangerous. It could cause cancer. This shows why it is so important that we carefully advance this technology.”