Editing the Human Genome

By: David Shifrin, PhD
Science Writer, Filament Life Science Communications

Two posts ago we looked at new advances in the CRISPR-Cas9 gene editing system. The DNA-snipping bacterial immune response has been adapted and adopted incredibly quickly by scientists. Arguably, CRISPR’s promise for everything from cell culture experiments in academic labs to curing human genetic diseases makes it one of the most potentially powerful biotechnologies of the past century.

Just because the technology is advancing rapidly doesn’t mean it’s ready to move out of animal models and into human clinical applications, though. This is true both from a biological standpoint and an ethical one. In fact, the rapid rise of CRISPR/Cas-9 gene editing means the ethical and regulatory considerations haven’t really had time to keep up. The idea of genetically engineering people has been around for years, but until CRISPR was discovered it remained nothing more than science fiction. Now, although the technology is not yet ready for application in humans (as opposed to human cells), CRISPR is arguably the first method that could realistically be used for human genome editing. This opens up a massive set of potential scientific, ethical, and political issues. In addition, while scientists are developing the technology and promoting its use in the lab, they are not necessarily in a good position to make the final decisions about its applications.

In this vein, Daniel Sarewitz wrote in a recent column over at Nature saying that, “Science can’t solve [CRISPR].” The main thrust of his argument is that scientists bring a defined and relatively narrow perspective to the debate about genome editing. This is not necessarily bad, but it does mean that scientists are not well positioned to deal with the social implications and, more significantly, societal concerns about the subject. This is where collaboration between science, the public, and policymakers comes into play. However, Sarewitz points out concerns that the scientific community isn’t taking the right steps for this to happen.

While prominent scientists have come out in favor of making “collective” decisions about advanced technologies like genome editing, scientific organizations are closing ranks for the discussions. Experts in various fields are coming together to talk about the issues, leaving representatives from the public conspicuously absent. According to Sarewitz, “The idea that the risks, benefits and ethical challenges of these emerging technologies are something to be decided by experts is wrong-headed, futile and self-defeating.”

That said, there are examples of collaboration between scientists and the public. These include the World Wide Views alliance and programs at NASA, according to Sarewitz. And, many scientists have been outspoken in their warnings for the scientific community to take its time in applying human gene editing. Another recent article, this one in Bloomberg Businessweek titled, “The Promise and Peril of Crispr [sic],” noted that the NIH will not award grants for human embryo gene editing. The scientific community as a whole is generally on board with this, “call[ing] for a moratorium on the use of Crispr in […] the human germline.” As exciting as the technology is, self-regulation among scientists and clinicians is critical to ensure that both gene editing and the public trust aren’t abused.

But again, it is not enough for scientists to self-regulate and just tell the public “we’ve got it under control.” Some level of transparency – seemingly one of this decade’s hottest terms – and lots of open engagement with politicians and the public is necessary to demonstrate what is being done to prevent abuse. This is especially true with the current state of CRISPR, where there is so much hype around a biotechnology that is so far from being applied in the clinic. The Bloomberg article notes that CRISPR-Cas9 gene editing doesn’t always work as hoped, and undesired mutations have occurred in some experiments. Being relatively open about those types of complications will go a long way in reassuring the public that scientists aren’t just plowing ahead, trying to create “frankenbabies.”

Another point that may help facilitate productive discussion of the topic (“productive” being the operative word) is this: the promise of CRISPR means that it is becoming a hot field for new commercial ventures. As a result, it seems likely that the increased money and attention flowing to gene-editing startups will only help grow the discussion about the technology itself. The more prominence (and VC funds) companies like Editas Medicine and CRISPR Therapeutics gain, the more the public will engage in the discussion and regulatory agencies will prioritize the field.

All told, CRISPR represents an incredible opportunity for science and medicine on multiple fronts. One can envision a future where disease-causing variants are revealed by genetic testing, and then the patient is instantly referred to a gene therapy lab down the hall for treatment. Personalized and precision medicine built to not just cure disease, but prevent it.

At the same time, the development of gene editing technology represents an opportunity for the scientific community to do something it has frequently struggled with: clearly communicating with various stakeholders on both the promise and reality of scientific research. Doing so will build trust, promote public support, and lead to an environment where new technologies can be implemented more effectively.