The peril and promise of gene editing
The claim on Monday by a Chinese scientist that he successfully edited the genetic makeup of a human embryo followed by live births is a moment for serious worry. Rapid advances in gene editing have been pointing toward this for a few years. If confirmed, the research must trigger a fresh re-examination of the procedures for research that could alter the basic blueprint of humankind. Vigilance, transparency and oversight are vital bulwarks against dangerous research, and this experiment appears to have skirted them all.
The researcher, He Jiankui of the Southern University of Science and Technology of China, in Shenzhen, said he carried out gene “surgery,” or editing, of embryos for seven couples during in vitro fertilization treatments. Using the technology known as CRISPR-Cas9, in which genetic material can be edited quickly and cheaply, He manipulated the embryo so as to disable a gene that opens the door to HIV to enter a cell. This deliberate mutation, if successful, would leave the person with genetic characteristics making them resistant to the virus that causes AIDS. He said one mother gave birth a few weeks ago to twin girls, Lulu and Nana, “as healthy as any other babies.” Their father carried HIV. He said Wednesday at a major scientific conference in Hong Kong he was proud of his work and that another pregnancy was in progress.
But tough questions are being asked about his secrecy and about his research, which was not subject to independent scientific peer review of the procedures and results. In China, such gene editing is prohibited under a ministerial instruction to fertility clinics, although, according to MIT’s Technology Review, the prohibition may not have the force of law. In the United States and much of Europe, it is prohibited.
The extraordinary promise of CRISPR is that genetic material can be modified to prevent or eliminate disease. But if the changes become heritable, they could have serious and unpredictable consequences through future generations.
Last year, a panel of the National Academies of Sciences, Engineering and Medicine issued a report saying that such genetic editing of human embryos, sperm and eggs — the “germ line” — should be confined to very narrow circumstances, including after preclinical work has clarified risks and benefits, and “only for compelling reasons and under strict oversight.”
This is a reasonable standard. Yet it does not appear He met these criteria of oversight and transparency; his work looks reckless. Certainly, alternatives for combating HIV/ AIDS exist other than to fiddle with the germ line. Also, some scientists fear CRISPR can trigger off-target effects, meaning that a simple snip and repair might do unanticipated damage to other genes. Moreover, gene editing to modify human intelligence or appearance would raise profound and deeply troubling ethical issues. In the end, this wonderful new technology must be supervised, regulated and carried out for the good of humanity, without mistakes, malicious intent or runaway experiments.