Jennifer Doudna Answers Questions on CRISPR, Gene Editing’s Future

Jennifer Doudna

JENNIFER DOUDNA SPEAKS AT HER 13 FEBRUARY PLENARY SESSION AT THE 2016 AAAS ANNUAL MEETING. | BOSTON ATLANTIC PHOTOGRAPHY

Last year, the genome-editing method CRISPR came to power as a technology poised to change everything from how we fight disease to whether we might finally be able to rid the world of the mosquito. The journal Science named CRISPR its Breakthrough of the Year, andTime magazine named one of its discoverers, University of California, Berkeley’s Jennifer Doudna, as one of the 100 most influential people in the world.

In her 13 February plenary talk at the 2016 AAAS Annual Meeting, Doudna took her audience through the technical breakthroughs that turned her “curiosity-driven science project” on how bacteria build immunity against viruses into the technology that can edit any gene easily, accurately, and inexpensively. Afterward with reporters, she reflected on the technology’s implications for bioethics, business, and her personal and professional path forward.

CRISPR’s powerful potential and ease of use—there are even DIY kits aimed at schoolchildren—brought the technology under intense scrutiny last year. Scientists and the public are now considering the ethics of a tool that might be used someday to edit the genes in the human germline (eggs and sperm) to create new characteristics that could be passed on to subsequent generations, or to correct diseased or otherwise “unwanted” genes.

Doudna said she considers it one of her responsibilities as a researcher to ensure that she educates people about the technology and listens to their questions and concerns about its use. She says one of her biggest fears is “waking up one morning and reading about the first CRISPR baby, and having that create a public backlash where people ban or regulators shut this down, and I think that could be very detrimental to the progress of the field.”

She agreed with other researchers that the technology will be used to edit human embryos, “but I think it’s important to be discussing under what circumstances we would want to do that.”

 

Last January, Doudna helped to gather a group of scientists in Napa, California to discuss how to move forward cautiously with CRISPR human genome editing. The meeting prompted an international gene-editing summit convened in December 2015 by the U.S. National Academy of Sciences. Its participants released a statement concluding that it would be “irresponsible” for researchers to move forward with clinical human germline editing until its risks and benefits were better known, and there was “broad societal consensus” on its use.

Doudna and others have learned much more in just the past two to three years about how CRISPR editing works at the molecular level, and how it can be delivered to specific cells and tissues. The technology is being refined and applied at a blistering pace, but she acknowledged that one of the biggest technical challenges remains finding the right gene or genes to edit to bring about a desired effect.

“We don’t know yet, what changes do you make, if you want to affect addiction or schizophrenia?” she said. “These are all potentially multigenic disorders that have a lot of genetic components. I think it’ll be quite a while before we can sort that out.”

2015 was also the year that CRISPR took center stage in an epic patent fight, one that Doudna admitted may take years to sort out. The uncertain outcome hasn’t scared the pharmaceutical and biotech companies she works with away from their plans for CRISPR, she said. “There’s such an excitement about the science that people feel it would be higher risk not to proceed with using it in various applications than to wait and try and sort out the patent situation first.”

Doudna said she receives email “daily” from people who want to learn more about whether CRISPR can be used to treat their diseases, or if there are any CRISPR clinical trials available, or if they can volunteer in some way to help with CRISPR-related research. She explains to most of them that CRISPR isn’t ready to tackle these problems yet. But she keeps a record of all the messages, she said, “and I want to answer all of them.”

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