Special Briefing
IN ASSOCIATION WITH THE PRESIDENTIAL DAILY BRIEF

Life Hack

The CRISPR Dossier

This is an OZY Special Briefing, an extension of the Presidential Daily Brief. The Special Briefing tells you what you need to know about an important issue, individual or story that is making news. Each one serves up an interesting selection of facts, opinions, images and videos in order to catch you up and vault you ahead.

WHAT TO KNOW

What happened? The scientific community exploded last week when Chinese scientist He Jiankui announced he’d successfully used CRISPR gene-editing technology on twin girls, tweaking a gene in order to make them resistant to HIV infection. He said the experiment, which hasn’t yet been peer-reviewed and was immediately denounced as unethical by most of his peers, made him “proud.” But there are dangers: Recent studies into CRISPR technology have found that gene-editing can trigger cancer or wipe out huge chunks of DNA that weren’t targeted.

Why does it matter? He Jiankui’s leap forward has brought a huge oversight into focus: There is no international regulatory framework governing the use of CRISPR technology, and many countries rely on scientists to self-regulate. Now some are questioning whether that’s enough — especially as He says a third edited baby may be on the way — and condemning He as irresponsible and unethical. But the public may not agree: A recent survey of 4,771 people in China found that 60 percent support research into, and the application of, gene-editing technology to treat diseases.

HOW TO THINK ABOUT IT

Nuts and bolts. CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, or chunks of recurring DNA that defend against viruses. But normally the term CRISPR refers to CRISPR-Cas9. Cas9 is a protein that slices through the DNA, and when that protein is injected into a cell it can snip out specific genes to disable them. Many in the medical community hope this can offer a workable way to fight against gene-based disorders like sickle cell anemia and cystic fibrosis. But editing genes can have unintended consequences — not just for the edited embryo, but also for their descendants, if the edited gene is passed on.

Global game. New experiments — and successes — involving genetic manipulation of human embryos have been happening around the world. The “three-parent” technique, which mixes DNA from three individuals to prevent deadly mutations being passed on, was legalized in the U.K. and first performed in 2016 by American scientists in Mexico. Since then a clinic in Ukraine has duplicated the feat. Meanwhile, Korean and Singaporean officials have granted CRISPR patents, German pharmaceutical companies like Bayer have invested hundreds of millions in potential CRISPR treatments and the French government has launched a project to share research on the technology. Outside of China, though, the technology hasn’t resulted in any live births. Meanwhile, other scientific issues requiring a global regulatory framework, like dealing with pandemics, offer few lessons for creating and enforcing regulations.

Ethical considerations. There’s been near-universal condemnation of gene-editing human embryos outside the scrutiny of regulatory bodies. When news broke about He’s work, more than 100 scientists signed a petition calling for greater oversight. China reiterated that gene-editing of human embryos for reproductive purposes is banned in the country. But some scientists are advancing CRISPR research in what may be more morally palatable ways, like editing adult genes to help alleviate disease. Patients with Hunter syndrome have shown promise after undergoing gene-editing treatment, which allows their livers to produce an enzyme curbed by the disease. Others hope CRISPR will be a valuable weapon in the fight against heritable conditions like diabetes, muscular dystrophy and Huntington’s.

Plants and animals. A great deal of research is going into using CRISPR in ways that don’t have to do with humans. Across the globe, companies are developing uses for CRISPR in agriculture, with edited corn DNA and trimmed animal embryos that they hope will breed healthier livestock. Scientists at the Institute for Sustainable Agriculture in Spain recently used CRISPR to edit wheat to make it safe for people with celiac disease, and last year scientists created a strain of algae using CRISPR technology that produced twice the normal amount of lipid, which can be used as biofuel. Meanwhile, the Bill & Melinda Gates Foundation has backed seven years of research in hopes of eradicating malaria by editing the genome of mosquitoes.

WHAT TO READ

Editing Babies? We Need to Learn a Lot More First, by Eric J. Topol in The New York Times
It is difficult to envision a foolproof way to rein in such rogue efforts, notwithstanding the international consensus that we are still nowhere near ready for genome-edited babies. Scientists like Dr. He should be castigated by their institutions and the biomedical community, as he was, and perhaps that will discourage this sort of unethical research.”

This Scientist Turned CEO Wants to Gene-Edit a Way to Cure Cancer, by Molly Fosco at OZY
"Working with cutting-edge biotechnology comes with an immense set of challenges. UC Berkeley and the University of Vienna are tied up in a legal battle with the Broad Institute of MIT and Harvard over the patent rights to CRISPR."

WHAT TO WATCH

What Happens When CRISPR Backfires?

“New research reveals that in about 20 percent of cells, CRISPR results in much larger deletions than we thought.”


About Lulu and Nana: Twin Girls Born Healthy After Gene Surgery as Single-Cell Embryos

“The gene surgery worked safely. No gene was changed except the one to prevent HIV infection. The girls are safe and as healthy as any other babies.”

WHAT TO SAY AT THE WATERCOOLER

Rule breaker. While China’s health ministry has called for an investigation into He’s use of CRISPR-Cas9, he’s only accused of breaking a 2003 guideline — one with no penalties attached. While he’s been ordered to stop his experiments, He says they were already on hold. As for a scientific investigation of whether his treatment actually worked, he’s promised a forthcoming paper, but there are still questions about how to properly peer-review the work while keeping the parents and children anonymous.