Gene Editing Could Stop Cancer, Diabetes and Bioterrorism: An Interview With CRISPR Scientist Jennifer Doudna

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French microbiologist Emmanuelle Charpentier (R) and professor Jennifer Doudna of the U.S. visit a painting exhibition by children about the genome, at the San Francisco park in Oviedo, October 21, 2015. Eloy Alonso/REUTERS

Earlier this week, a team of scientists, led by a researcher at Oregon Health and Science University, published a paper showing it’s possible to alter human embryo DNA to prevent congenital disease. The study shows that CRISPR-Cas9 is certainly powerful. But in the fanfare and controversy surrounding the news, the public may have lost sight that CRISPR is also highly versatile.  

Scientists are using the technology to develop effective treatment therapies for a range of diseases, including cancer, diabetes and communicable diseases. Other researchers apply gene editing to solve agricultural problems, counter bioterrorism and clean up the environment.

Since CRISPR was first identified, geneticists have been adapting it in the laboratory as a tool that could be used to alter genetic codes of all living organisms. The study, published in Nature on Wednesday has incited a debate about the ethics of using CRISPR technology to alter human genes, which draws attention to the ongoing public fear that humanity will soon have the capacity to build designer babies.

Newsweek spoke with Jennifer Doudna, a microbiologist at the University of California, Berkeley and co-discover of the breakthrough gene-editing technique, about how quickly the technology is advancing and the progress she expects to see in the future.

What do you make of the findings in the Nature study?

In a way it’s not a surprising study. There’s obviously been interest in the potential application of genome editing to curing genetic disease. Ultimately, if one could do this in the germline, it would be possible to get rid of disease-causing mutations at the beginning of life.

What’s really interesting here is that the study was conducted in a way that could create a path to the clinic, and to establish a procedure for doing gene editing that would be feasible in these embryos. The researchers largely achieved that.  

What’s the one thing you say to people to try to assuage their the worries that we’re on the path to creating designer babies?

People say it won’t happen in the U.S. but what about China? I am asked this question at cocktail parties. What about Asia? What about places that have fewer restrictions, and perhaps fewer cultural feelings against germline editing? It’s entirely possible that there will be use of germline editing in those jurisdictions. I encourage the scientific and clinical communities around the world to not rush CRISPR to clinical research because I think it would be a shame if a powerful technology gets a black eye in the public perception, at least in terms of using it inappropriately.

Are there other ways to use this technology in a reproductive medicine setting that don’t involve editing an actual embryo?

Perhaps in the not-too-distant future it will be possible to generate gametes—meaning eggs or sperm—from somatic cells in a person. Already it is possible to do this in animals. Once this is technically feasible in humans, doctors could use CRISPR for patients with a known genetic predisposition to something or certain mutations to generate gametes that could be used in an in vitro fertilization setting. This removes the issue of embryo editing, though it doesn’t remove the issue of making changes that become heritable in the human germline.

Are you surprised by how fast this research has progressed?

It’s been about five years since we published our paper describing the CRISPR system and how it could be used for genome editing. I never imagined back then that I would be reading this headline in the New York Times this week.

What are you working on that shows CRISPR’s broad capabilities?

I’m leading the Innovative Genomics Institute, a UC San Francisco partnership aiming to bring genome editing to important problems in human health and the environment, which is aimed at bringing people who do fundamental research like me together with clinicians and plant biologists. We’ve teamed up with neurosurgeons at UC San Francisco, and we’re developing ways to deliver gene-editing molecules into the brain. This has nothing to do with germline editing. This is therapy for neurological disease. I’m very excited about the potential to use gene editing to correct mutations that could really benefit patients in the future.

We published a paper in Nature Biotechnology earlier this year showing how we can use CRISPR for editing DNA in the brains of mice. We’re focused right now on Huntington’s disease and working in a couple of different animal models to investigate whether the approach has a therapeutic benefit in these animals. If that looks promising then we hope to make steps toward clinical trials with our partners at UCSF.

The vast majority of scientists right now who are working with gene editing—and CRISPR in particular—are focused on this type of application. Researchers are not trying to make heritable changes to DNA in humans. They are trying to make changes to DNA that would impact a patient in their lifetime and have a positive effect.