How far should we go with gene editing in pursuit of the "perfect" human?

February 05, 2023

The Third International Summit on Human Genome Editing, which will take place at the Francis Crick Institute in London next month, does not identify He Jiankui as a registered delegate. However, the majority of attendees will be thinking about the disgraced Chinese scientist. He'll be a ghost at the science feast.

Jiankui was accountable for one of the most contentious events in contemporary science, as was made public during the previous global summit on genome editing, which took place in Hong Kong in 2018. The researcher, who was then working at China's Southern University of Science and Technology in Shenzhen, revealed to the delegation that he had altered the genetic makeup of three young girls in an effort to make them immune to HIV. Future generations would then be able to inherit this change, which was made when they were embryos.

Chinese officials found the experiment unethical and without precedence in contemporary genetics. Jiankui was later sentenced to three years in prison, but according to Professor Robin Lovell-Badge, the event's organiser, his impact at the upcoming science summit in London will still be significant.

"We will be discussing what has happened to the three children whose physiology he may have altered by genome editing," said Lovell-Badge, who also chaired the session at which Jiankui revealed his extraordinary biological intervention. "We will also have presentations about the changes that have occurred in China in terms of the law and the ethics governing gene editing." There have clearly been quite substantial changes—for the better.

"And it is crucial that these topics are brought up." Before the technology is put into use, we should be open and honest about how genome editing is being tested and evaluated for human benefit.

Jennifer Doudna of the University of California, Berkeley, and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology in Berlin revolutionised genome editing. In 2020, the Royal Swedish Academy of Sciences, which presented the award, recognised the duo's work by awarding them the Nobel prize in chemistry for developing "a technology [that] has transformed the molecular life sciences."

The method created by Doudna and Charpentier is called Crispr-Cas9, and it functions like a pair of molecular scissors that can cut a strand of DNA at a particular spot. By changing the genetic makeup of plants, animals, and people, scientists can affect physical characteristics like eye colour and disease risk. A key distinction from earlier types of genetic manipulation is that no genes from other organisms are introduced.

Scientists are currently investigating genome editing to provide novel medical therapies, such as altering those who already have disorders. Candidates include sickle cell anaemia, an inherited disorder in which a single gene error impairs the manufacturing of haemoglobin with catastrophic repercussions for patients who experience anaemia as a result of their bodies being starved of oxygen.

Red blood cells can be returned to a person's body by extracting their stem cells and genetically modifying them to begin producing foetal haemoglobin, a process that is typically turned off at birth. Several centres are already conducting trials.

Additionally, medical professionals and researchers are looking into how to employ genome editing to treat a variety of crippling diseases that have resisted previous attempts to treat them, including muscular dystrophy, cancer, diabetes, some forms of congenital blindness, and many others. Numerous delegates will attend the summit the following month to learn about the most recent developments.

You might even consider altering liver enzymes to improve how well both men and women can detoxify their systems from chemical warfare toxins or to increase their resistance to biological weapons, continued Lovell-Badge. The kind of human augmentation that military researchers are currently considering is that.

You might also think about modifying people so they can see infrared or ultraviolet light, like certain animals can. These improvements would be perfect for soldiers engaged in combat at night or under other adverse circumstances.

The extent to which society will accept such human upgrades is a different issue, one that will be covered at a subsequent Francis Crick Institute event. Cut + Paste, a public exhibition, will investigate what alterations can be made to people safely using genome-editing technology, which ones ought to be ranked as priority, and which ones might be viewed as unethical and left out of further investigation.

According to Ruth Garde, curator of Cut + Paste, which debuts this week, "genome editing tools have enormous potential to improve human health and the environment, but like all new technology, they present ethical challenges and concerns." "At the moment, the public is not very aware of these procedures." Through a variety of interactive experiences, "Cut + Paste will enable visitors to study and reflect on the ethics of genome editing."

The exhibition will cover every facet of genome editing, including how it may be used to enhance crops and farm animals, but as with the upcoming international meeting, the main emphasis will be on how it affects people. Garde continued, "Genome editing has made it simpler to conceive "enhancing" human features. Visitors are prompted to consider what "improvement" entails by Cut + Paste. Who determines what qualifies as a "desirable" trait?

Visitors will be asked to consider a variety of applications for genome editing, including the ability to make mosquitoes infertile in order to combat diseases like malaria, the ability to improve human capabilities, and the ability to alter physiological traits that will be passed down through generations. Critically, we want visitors to express their opinions to us, Garde stated.

At the summit, these concerns will also be hotly debated. Lovell-Badge stated that while genome editing for sickle cell anaemia has great potential, it is also quite expensive. "One person's therapy may cost a million dollars." However, the continent with the least access to pricey medications is Africa, where the disease is most common. Is it therefore possible that we will widen the gap between developed and developing countries' levels of health?

The erratic behaviour of Jiankui, who later admitted acting "very soon," will heighten the tension in the conversation. Jiankui is currently out of prison and operating a laboratory once more in Beijing, according to Lovell-Badge. He claims that he will concentrate on gene therapy to address conditions like muscular dystrophy. He is not a biologist, and that bothers me because he knows little about the illness.

The scientist has now mentioned how Jiankui's prior acts may have been motivated by the possibility that having HIV can make one an outcast. He intended to avoid that. He decided to attempt altering the CCR5 gene, which has innate variations that can guard against HIV. He intended to produce genetically modified protection in this manner, according to Lovell-Badge.

However, research has also revealed that in 20% of instances, these genome-editing modifications might result in significant rearrangements of a person's genome, which is extremely risky. "This underscores why it is so vital that we move this technology forward carefully," the scientist said.