SciTech

First babies with (maybe) edited genes born

For better or for worse, Chinese geneticist He Jiankui may have just opened the Pandora’s box of gene-editing.

He, a researcher based in Shenzhen, China, claims to have created the world’s first genetically edited babies. “Two beautiful little Chinese girls, named Lulu and Nana, came crying into this world as healthy as any other babies,” said He in a YouTube video posted on his company’s channel on Monday. Before being implanted in the womb as embryos, Lulu and Nana’s genes were altered to prevent contraction of HIV. He used CRISPR-Cas9 technology to perform gene surgery in order to reproduce a natural genetic variation that confers strong resistance to HIV-1 infection.

He’s actions have caused a global outcry, with scientists worldwide expressing shock and outrage. More than 120 Chinese scientists issued a joint statement on Monday, condemning He’s use of the CRISPR-Cas9 on a baby as a “huge blow” to the ethical standing of Chinese biomedical research. Dr. Eric Topol, who heads the Scripps Research Translational Institute in California, described the experiment as being “far too premature.”

“We’re dealing with the operating instructions of a human being,” Topol explains. “It’s a big deal.”

A few scientists have spoken out against the criticisms, however. George Church, a famed geneticist at Harvard University, argued that He’s research is “justifiable” as a valiant attempt to battle HIV, “a major and growing public health threat.”

The Chinese government has halted the work of He’s lab, having ordered immediate investigations into the experiments. Furthermore, Professor He’s university, the Southern University of Science and Technology in Shenzhen, said it was unaware of the research project and that Professor He has been on unpaid leave for the past few months. Gene editing outside of lab research is outlawed or restricted in many countries, including China.

The CRISPR gene editing technique used by He was developed in 2013, and while its potential has been widely praised, it is still considered experimental. Many unintended mutations, capable of causing genetic problems later in life, including cancer, are associated with the procedure. As explained by Feng Zhang, one of the key inventors of CRISPR, “when you change one thing, something else gets changed, too.”

Several scientists have reviewed the experimental materials that He provided to the public and said the tests are insufficient to confirm whether the editing worked or caused harm.

It was also noted that the evidence may suggest that the editing was incomplete and left various changes to a patchwork of cells. Moreover, even if editing worked perfectly, the imposed HIV-resistant gene variation is known to increase risk of other viruses and diseases. Such evidence begs the question of how much there was to be gained in terms of protection against HIV.

Professor He’s lab has altered embryos for over seven couples, but only one of the experiments has led to pregnancy. Many are unsettled that the participants may not have given fully informed consent to the treatment, as He’s consent forms called the project an “AIDS vaccine development” program.

However, He maintains that the patients were well aware of the nature of the research, saying that he personally explained the forms to each family “line by line.” He also promised to provide insurance coverage for any children conceived through the project and medical follow-up until the children reach adulthood.

According to He, the parents involved refuse to be identified or interviewed. He also would not disclose where the parents are from or where the work was done.

Professor He recognized early on that his work would cause much controversy. However, he believes “families need this technology,” He explained.
If the treatment causes unwanted side effects or harm, “I would feel the same pain as they do and it’s going to be my own responsibility.”