Gene Editing: The Good, the Bad, and the Ugly

In the 2016 Worldwide Threat Assessment of the US National Intelligence Community, Director of National Intelligence James R. Clapper noted: “given the broad distribution, low cost, and accelerated pace of development of this dual-use technology [genome editing], its deliberate or unintentional misuse might lead to far-reaching economic and national security implications.”

The CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats) gene-editing technique has enabled low-cost scientific breakthroughs, while simultaneously opening the door for possible misuse by malevolent actors. While advocating the need for strategic planning to account for the threat posed by gene editing, Mathew Burrows, director of the Strategic Foresight Initiative at the Atlantic Council, said, “the speed of these scientific developments…continues to outpace the ability for us to prepare.”

CRISPR-Cas9 has improved the gene editing technology that has been around since 1975. Consequently, “the technique is easy, it’s cheap, and it’s accessible, and if it falls in the wrong hands, like a terrorist group or rogue nations, it is possible for them to use the technique and develop weapons with more ease than twenty years ago,” said Dr. Pierre Noel, director of the blood and marrow transplant program in the division of hematology and professor of medicine at the Mayo Clinic in Arizona.

Noel, a nonresident senior fellow at the Atlantic Council’s Brent Scowcroft Center on International Security, moderated a panel discussion among medical and scientific professionals at the Atlantic Council on September 27 to examine the severity of the threat posed by gene editing and consider the strategic planning involved in mitigating the risk.

Other participants on the panel included Dr. Keith Stewart, director and professor of medicine at the Mayo Clinic Center for Individualized Medicine; Col. Nelson Michael, director of the US Military HIV Research Program at the Walter Reed Army Institute of Research and professor of medicine at the Uniformed Services University; and Paula Bryant, senior scientific officer at the Office of Biodefense Research Resources and Translational Research for the National Institute of Allergy and Infectious Diseases.

The panelists agreed that the science of gene editing alone is useful, even life-saving, yet the potential misapplication makes the technology a threat.

According to Stewart, “for twenty years we’ve had the ability to genetically manipulate human cells and other materials,” such as agricultural products modified for enhanced yield or livestock fortified against disease. Stewart referred to the genetic manipulation of human cells in gene therapy, conducted on an individual basis with the benevolent intent to cure disease. However, in 2015, Chinese scientists published for the first time a report on their experience gene editing in a human embryo, an instance of “germline editing” not confined to an individual’s cells but a transformation that can be transmitted through generations.

This development served as a “major wake-up call” to the international community, according to Noel. Though rudimentary and unsuccessful, the Chinese experiment raised alarm as to the potential applications of the technology. Noel said, “it’s possible that in the future, as the technology becomes more sophisticated,” countries may be able to implement gene-editing technology to design what he called “super soldiers.” Stewart described how “you could, at least in principle, make…a soldier with great muscle force and strength.”

“We began to be a little bit concerned about the oversight and control of…human embryo gene editing,” said Stewart. The work of the Chinese scientists prompted an international summit to discuss the ethics and regulation of the technique.

In 2010, when synthetic biology first became a prominent practice, US President Barack Obama asked the Presidential Commission for the Study of Bioethical Issues to review the field, particularly, according to Michael, “to consider the potential medical, environmental, security, and other benefits, as well as potential health, security, or other risks.” Michael served on the committee and said the recommendations proposed in the 2010 report could serve as a helpful framework for considering the regulation of human genome editing with the intent of mitigating associated threats to national security. In particular, he said that the recommendations for public beneficence and responsible stewardship could play a role “in context with other ongoing debates about bioethics for gene editing.”

According to Bryant, “there’s ethics around the use of [CRISPR-Cas9], but obviously someone in a terrorist organization or a do-it-yourselfer may not follow those same ethics as they approach it.”

When used with prudent regulation and responsible use of the technology, CRISPR-Cas9 has great research benefits, Bryant said. She described how the gene-editing technique has been used for gene therapy, diagnostic development, and vaccine development to combat harmful pathogens such as the Zika virus. Gene-editing has also been used in trials to treat certain types of cancer within the afflicted patient’s cells, with success. Bryant claimed that CRISPR “brings a lot of good to the biodefense community with regard to making medical counter-measures.”

However, the dual-use aspect of gene editing poses a significant risk. “There’s this concern that if it benefits us, it’s going to make it that much easier for someone who has a nefarious intent” to threaten US national security, according the Bryant. “In biodefense, we have to prepare for the unknown,” she claimed.

Right now, “we have a generation of do-it-yourself gene hackers who go either in community labs or in their garages or in their kitchens and are able to use CRISPR for gene editing,” said Noel. Due to the sophistication and extensive resources required for more complex projects, Noel said, “at this point in time, the editing of microbes and creating of bioweapons is felt to be out of reach of do-it-yourself gene hackers.”

However, “the ease of the technique opens the door for other individuals, maybe a little bit more sophisticated, of producing bioweapons,” said Noel.

“The technology is out there,” said Bryant, and this “changes the face of the threat.”

When considering what the biodefense community needs to protect against, Bryan said, “it’s not just the pathogen, it’s also who’s going to release that pathogen or use it in a bad way because that helps define the threat.” She asserted that capable and suitably-equipped actors who pose a security threat require malicious intent, sufficient financial means, access to pathogens, and ability to transform these pathogens into a weapon. “If you start thinking about who has access to what,” said Bryant, “the state actor seems to be the primary player.”

“From the biodefense perspective…we have always been concerned about that unknown genetically-engineered threat,” Bryant said. She said CRISPR-Cas9 “makes the threat more realistic and validates that we were concerned about it, because yes, it could possibly be done.”

Michael described how CRISPR and gene editing, should the technology fulfill its potential, could give terrorist groups or other malevolent actors “the opportunity to make one hundred different variants [of a genetically-engineered threat] in the time it would [currently] take to make one, and the consequences of having one hundred variants out there, maybe one slips through and is lethal, and is persistent in the environment.”

“I think that’s the real threat,” he added.

Rachel Ansley is an editorial assistant at the Atlantic Council.

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Image: (from left) Dr. Pierre Noel, director of the blood and marrow transplant program in the division of hematology, professor of medicine at the Mayo Clinic in Arizona, and a nonresident senior fellow at the Atlantic Council’s Brent Scowcroft Center on International Security, moderated a panel discussion with Paula Bryant, senior scientific officer at the Office of Biodefense Research Resources and Translational Research for the National Institute of Allergy and Infectious Diseases; Col. Nelson Michael, director of the US Military HIV Research Program at the Walter Reed Army Institute of Research and professor of medicine at the Uniformed Services University; and Dr. Keith Stewart, director and professor of medicine at the Mayo Clinic Center for Individualized Medicine. (Atlantic Council)