Houston, Texas, USA : A scientist in China claims to have created the world’s first genetically edited babies, in a potentially ground-breaking and controversial medical first.
If true, it would be a profound leap of science and ethics. This kind of gene editing is banned in most countries as the technology is still experimental and DNA changes can pass to future generations, potentially with unforeseen side-effects.
Many mainstream scientists think it is too unsafe to try, and some denounced the Chinese report as human experimentation.
The researcher, He Jiankui of Southern University of Science and Technology in Shenzhen, said he altered embryos for seven couples during fertility treatments, with one pregnancy resulting so far. He said his goal was not to cure or prevent an inherited disease, but to try to bestow a trait that few people naturally have: an ability to resist possible future infection with HIV.
He said the parents involved declined to be identified or interviewed, and he would not say where they lived or where the work was done. There is no independent confirmation of He’s claim, and it has not been published in a journal, where it would be vetted by other experts.
He revealed it on Monday in Hong Kong to one of the organisers of an international conference on gene editing that is due to begin on Tuesday, and earlier in interviews with the Associated Press.
“I feel a strong responsibility that it’s not just to make a first, but also make it an example,” He said. “Society will decide what to do next” in terms of allowing or forbidding such science.
Some scientists were astounded to hear of the claim and strongly condemned it. It was “unconscionable … an experiment on human beings that is not morally or ethically defensible,” said Dr Kiran Musunuru, a University of Pennsylvania gene-editing expert.
“If true, this experiment is monstrous,” said Julian Savulescu, a professor of practical ethics at the University of Oxford. “The embryos were healthy. No known diseases. Gene editing itself is experimental and is still associated with off-target mutations, capable of causing genetic problems early and later in life, including the development of cancer.”
“There are many effective ways to prevent HIV in healthy individuals: for example, protected sex. And there are effective treatments if one does contract it. This experiment exposes healthy normal children to risks of gene editing for no real necessary benefit. In many other places in the world, this would be illegal punishable by imprisonment.”
In recent years, scientists have discovered a relatively easy way to edit genes, the strands of DNA that govern the body. The tool, called Crispr-Cas9, makes it possible to operate on DNA to supply a needed gene or disable one that is causing problems.
It has only recently been tried in adults to treat deadly diseases, and the changes are confined to that person. If sperm, eggs or embryos were to be edited, the changes could then be inherited.
He Jiankui studied at Rice and Stanford universities in the US before returning to his homeland to open a lab at Southern University of Science and Technology of China in Shenzhen, where he also has two genetics companies.
He said he practised editing mice, monkey and human embryos in the lab for several years and has applied for patents on his methods. He said he chose embryo gene editing for HIV because these infections are a major problem in China. He sought to disable a gene called CCR5 that forms a protein doorway that allows HIV, the virus that causes Aids, to enter a cell.
All of the men in the project had HIV and all of the women did not, but the gene editing was not aimed at preventing the small risk of transmission, he said. The fathers had their infections deeply suppressed by standard HIV medicines and there are simple ways to keep them from infecting offspring that do not involve altering genes. Instead, the appeal was to offer couples affected by HIV a chance to have a child that might be protected from a similar fate.
He said the gene editing occurred during in vitro fertilisation. First, sperm was “washed” to separate it from semen, in which HIV can lurk. A single sperm was placed into a single egg to create an embryo. Then the gene-editing tool was added. When the embryos were three to five days old, a few cells were removed and checked for editing. Couples could choose whether to use edited or unedited embryos for pregnancy attempts. In all, 16 of 22 embryos were edited, and 11 embryos were used in six implant attempts before the twin pregnancy was achieved, He said.
Tests suggest that one twin had both copies of the intended gene altered and the other twin had just one altered, with no immediate evidence of harm to other genes, He said. People with one copy of the gene can still get HIV.
Musunuru said that even if editing worked perfectly, people without normal CCR5 genes faced higher risks of contracting certain other viruses, such as West Nile, and of dying from flu. Since there are many ways to prevent HIV infection and it is treatable if it occurs, those other medical risks are a concern.
There also are questions about the way He said he proceeded. He gave official notice of his work long after he said he started it, on 8 November. It is also unclear whether participants fully understood the purpose and potential risks and benefits; for example, consent forms called the project an Aids vaccine development programme.
He said he personally made the goals clear and told participants that embryo gene editing had never been tried before and carried risks. He said he also would provide insurance coverage for any children conceived through the project and plans medical follow-up until the children are 18, and longer if they agree once they are adults.
“I believe this is going to help the families and their children,” He said. If it caused unwanted side-effects or harm, “I would feel the same pain as they do and it’s going to be my own responsibility”.
Dr Sarah Chan, a bioethicist at the University of Edinburgh, said that if true, the experiment was “of grave ethical concern”.
“Whether or not the veracity of these reports is eventually borne out, making such claims in a way that seems deliberately designed to provoke maximum controversy and shock value is irresponsible and unethical,” she said.
“The claim made by those responsible for the research is that the babies have been genome edited in an attempt to make them immune to HIV. The lifetime risk of contracting HIV is extremely low in the first place; there are other means of prevention and it is no longer an incurable, inevitably terminal disease. Putting these children at such drastic risk for such a marginal gain is unjustifiable.”
Q&A on scientist’s claim of gene-edited babies
Designer babies might be here sooner than anyone reckoned. A Chinese researcher who says he created gene-edited babies crossed what most scientists consider a forbidden line.
It’s not clear if the claim is true and if so, how the twin girls whose DNA reportedly was altered will fare as they grow.
There is wide scientific agreement that rewriting DNA before birth—to prevent an inherited disease or to give a baby some “designer” trait—isn’t yet safe to try outside laboratory experiments that do not lead to human births.
“Grossly premature and deeply unethical,” is how noted U.S. bioethicist Henry Greely of Stanford University characterized the claim.
The researcher, He Jiankui of Shenzhen, said he altered embryos when parents were undergoing fertility treatments to change a gene so that it might provide the resulting babies with a trait few people naturally have—protection against future infection with the AIDS virus.
“This is probably the worst gene you would choose” to test in pregnancy because it doesn’t fix a disease the children were destined to get, said Shoukhrat Mitalipov of the Oregon Health & Science University, who in laboratory-only experiments studies how to repair gene defects in embryos.
“Where is the assurance this mutation now will result in resistance to HIV?” Mitalipov added. “He’s testing his hypothesis on babies.”
Here are questions and answers about Monday’s claim and the state of gene editing:
What Is Gene Editing?
It’s a technology that lets scientists alter the DNA of living cells—from plants, animals, even humans—more precisely than ever before. It’s like a biological cut-and-paste program: An enzyme that acts like molecular scissors snips a section of a gene, allowing scientists to delete, repair or replace it.
How Is Gene Editing Used?
Researchers routinely use gene-editing tools in labs to study diseases in cells or animals, and they’re altering crops and food animals for better agriculture.
But in people, gene editing still is highly experimental. One first-in-human study is testing intravenous infusion of gene-editing ingredients to fight a killer metabolic disease. Other researchers are developing ways to gene-edit damaged cells and return them, repaired, into patients with sickle cell disease and other disorders. But unlike Monday’s announcement, none of those experiments would alter DNA in a way that patients would pass to their own children.
What Did The Chinese Scientist Do?
The researcher said he used the gene-editing tool CRISPR to alter a gene named CCR5 in embryos for seven couples during their fertility treatments; one pregnancy resulted. A particular mutation in that CCR5 gene is thought to confer some resistance to HIV by making it harder for that virus to enter cells.
Today’s medications have turned HIV from a death sentence into a manageable disease in much of the world, but He said he chose that gene because HIV remains a big problem in China.
But He’s claims have not been verified by outside scientists, and there are questions about how the work was conducted.
Why Is Monday’s News So Controversial?
Altering genes in sperm, eggs or embryos means those changes can be passed down to future generations—people who would have no way to consent to those changes. Plus, long-term negative effects might not become apparent for years.
In 2017, the U.S. National Academies of Science, Engineering and Medicine said lab-only research to learn how to alter embryos is ethical—but said it’s not ready for pregnancies yet. The academy said if it is ever allowed, it should be reserved to treat or prevent serious diseases with no good alternatives.
That lab-only research is going on, by Mitalipov and others.
But critics said Monday’s announcement opens the door to “designer babies.”
“If this goes unchallenged, other rogue actors will soon offer wealthy parents purported genetic enhancements for their children,” said Marcy Darnovsky of the Center for Genetics and Society.
What Happened To The Babies?
No independent outsiders know yet, which is partly why scientists are so disturbed.
He, the Chinese researcher, said one twin had both copies of the intended gene altered while the other had just one altered. People with one copy of the mutation can still get HIV.
Scientists who reviewed his claims said the alterations aren’t an exact match to natural CCR5 mutations, and that a big question is whether the gene is altered in every cell.
The particular method used is common in lab research but not precise or controlled enough for embryos, said Columbia University cell biologist Dietrich Egli, who called it “essentially genome vandalism.”
What Are The Dangers?
The biggest concern: That precision, or lack of it. Unintended mutations could harm health rather than help it.
Is Gene-editing For Pregnancy Legal?
Where you live determines if, or what kind of, research can be performed on human embryos. In the U.S., scientists can perform laboratory embryo research only with private funding, not with federal taxpayer money. Any pregnancy attempt would require permission from the Food and Drug Administration, which is currently prohibited by Congress from even reviewing such a request—a de facto ban.
Are There Other Ways To Prevent Inherited Diseases?
People undergoing fertility treatments that include IVF can have embryos tested for deadly gene mutations that run in the family, such as Huntington’s disease, and then implant only the embryos that lack such mutations. Also, some so-called mitochondrial disorders can be addressed by using some genetic material from mom and some from a donor egg, along with dad’s sperm.
Progress in genetic testing of embryos stokes fears of designer babies
Recent announcements by two biotechnology companies have stoked fears that designer babies could soon be an option for those who can afford to pick and choose which features they want for their offspring. The companies, MyOme and Genomic Prediction, have been working on technology that they hope to sell to fertility clinics, which could someday lead to the option of terminating pregnancies if fetuses have undesirable characteristics, such as low IQ levels.
Genetic testing of embryos fertilized via in vitro fertilization has been available for almost 30 years—it is routinely conducted to look for abnormalities in chromosomes that lead to disorders such as cystic fibrosis. But such tests have been limited in scope. Advances in technology now allow for screening for more possible conditions and soon, certain undesirable characteristics.
More specifically, there are two new options that will soon be made available to parents considering IVF. The first involves obtaining genetic samples from both parents and the embryo, and using a computer algorithm to come up with a more complete chromosomal profile for the embryo. The second technology involves sequencing the embryo’s DNA and subjecting the results to machine learning algorithms that are able to identify patterns among DNA elements that suggest the likelihood of a person developing some types of diseases, such as certain cancers.
The new technology gives prospective parents more leeway in deciding whether to implant an embryo and allowing it to grow and eventually to be born. But it also opens the door to more difficult decisions in the future, as biotechnology continues to improve. Researchers at such companies as MyOme and Genomic Prediction have mentioned that it might soon be possible to make a better-than-average prediction of intelligence based on genetic markers. That poses the question of whether it is unethical to filter for intelligence or other non-health related characteristics, such as eye or hair color—or perhaps someday, how good-looking a child will be, or how personable.
Officials for both companies have been careful to point out that they will only be offering testing for diseases, at least for now. Critics have suggested that it is just a matter of time, however, before designer-baby options become available.