In first Lemley lecture, CRISPR-Cas9 co-inventor explores the science, ethics of genome editing

In a virtual presentation at Brown, pioneering biochemist Jennifer Doudna discussed the intellectual curiosity that led to the development of the CRISPR-Cas9 technology, its electrifying promise — and the ethical questions it poses.

PROVIDENCE, R.I. [Brown University] — When Jennifer Doudna, a professor in the departments of chemistry and of molecular and cell biology at the University of California, Berkeley, found out that she had won the Nobel Prize, her husband had some news for her. 

“You are truly an ambassador of science now,” Jamie Cate, Doudna’s husband and a fellow scientist and professor at U.C. Berkeley, told her. “Your job, more than anything else, is to speak about science in a public way and to really communicate science to people who are non-specialists.”

Doudna shared that anecdote during a Thursday, March 4, virtual presentation to the Brown University community, titled “The CRISPR Revolution: The Power & Promise of Gene Editing,” the inaugural installment in the Lemley Family Leadership Lecture Series

Throughout the event, she untangled for the audience the complexities of the science that led to the development of the groundbreaking genome-editing technique CRISPR-Cas9 — what ultimately earned Doudna, along with her collaborator Emmanuelle Charpentier, the 2020 Nobel Prize in Chemistry. 

“This is a tool that allows genome editing,” Doudna explained. “What that means is that it’s a way to change the sequence of DNA in cells in a precise fashion.” 

She shared a representation of a Cas-9 protein cutting the double-stranded DNA — the code of life. 

“It literally functions like chemical scissors that can cut DNA at a position that can be programmed by the scientists.” The cell can then repair that break, which may involve introducing a new change into the DNA sequence, altering the code.

The technology was discovered almost incidentally, Doudna said, through research to understand how bacteria fight viral infection. 

“This is a kind of a great shout-out to what I like to call ‘small science,’ which is science going on in just a handful of labs around the world,” Doudna said. “It was really about our curiosity about how an immune system might operate in bacteria — something that, until about two decades ago, nobody knew such a thing even existed.”

Doudna gave many other shout-outs, as well, to the scientists whose research, ideas and questions led to CRISPR and continues to inform its applications. In just eight years, the technique has become almost commonplace. 

“For the students attending this,” Doudna said, “I would say that if you're studying any area of biology, you will probably encounter CRISPR.” We’re now in an “era of genome editing,” Doudna noted, in which the technology is used widely in medicine as well as agriculture. 

Brown President Christina H. Paxson, who moderated the event, asked Doudna to comment on the scope of CRISPR as a cure. Were there any areas where this technology doesn’t make sense, she asked? 

Doudna said that CRISPR is already being used to treat sickle cell anemia — although, she added, more work needs to be done to increase accessibility and affordability — and she saw promise for treating similar diseases in which altering a single gene could result in beneficial changes. She predicted therapies for multiple sclerosis and potentially cystic fibrosis. Further into the future, she said, CRISPR might be used to treat more complex diseases like Alzheimer’s, schizophrenia and, eventually, cancer. 

“I'm optimistic that it will be possible to use CRISPR together with immunology therapies to find ways to program the immune system to be more effective against cancer,” she said. 

With the COVID-19 pandemic continuing to impact individuals and communities across the world, Doudna said she is excited now about using CRISPR as a molecular diagnostic tool to detect COVID-19. Her lab is working with a large academic consortium to develop a test that could be used on-site to quickly and accurately test for asymptomatic virtual infection. 

In addition to highlighting the promises of genome editing, Doudna spoke about some of the ethical questions related to specific applications — like germline editing — in which the technology could create genetic changes that are passed down through generations. She acknowledged that earlier in her career, she focused on science and left ethics to others. 

“I never imagined that as a scientist, I would have any need to get involved in a public discussion around the ethics of science,” she said. “With CRISPR, it was pretty much immediately clear there were going to be some really big, really important implications of the technology.” 

She initially felt self-conscious, she said: “I thought: Who am I to be speaking publicly about this? I don't know what I'm talking about. I realized nobody else does, either, because the technology is brand new.” 

Doudna said she now stresses the importance of discussing ethical dilemmas in a way that’s informed by transparency, expertise and perspectives from individuals from multiple fields of study. 

“CRISPR might sound scary,” she said near the end of the event. “I think what is important to appreciate is that it requires a lot of expertise to use it... I think what is more important is to appreciate how the technology is going to enable more knowledge in the future, and how we need to be aware of the potential of the technology.

“Not that we need to be afraid of it. But we need to deal with it responsibly.”

Class of 1969 graduate Karen Sorkin Jakes, whose undergraduate experience at Brown inspired a career as a research molecular biologist, tuned in virtually to watch the inaugural Lemley lecture with her husband, a Class of 1968 alumnus.  

“It was fabulous! The science was clear and I think accessible to a more general audience, and, of course, the subject matter couldn't be more important or exciting,” Jakes said. “And Dr. Doudna's comments about the obstacles to scientists, particularly women, trying to get their labs up and running at the very point in their lives when they have young children and possibly also aging parents to care for, were particularly thoughtful... It was a gem and I'm so grateful to have been able to see it.”