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has led to such rapid uptake by the research comm
unity. Researchers can learn to use it and adapt it to their own area of study in almost no time. The huge potential for medical purposes makes the technique a real game changer. “There are so many unmet rare genetic diseases and a tool like CRISPR gives us a way to address them by engineering personalized treatments at scale. We still largely approach medical research as “one disease, one cure”, but genome editing can be a platform technology that allows us to treat whole classes of diseases with quick, minor alterations,” says Doudna. Currently the biggest challenge with the CRISPR therapy is delivery of gene editors to target tissues, says Doudna. “We know a lot about different disease targets, but in many cases we can’t get the gene editors to those cells in vivo.” This is an area her own lab and several other labs at the Innovative Genomics Institute are focused on, since solving the delivery challenge is key to opening up a world of potential treatments. Making the therapy affordable for patients is also a main hurdle to cross. Doudna takes Sickle Cell Disease as an example: The current CRISPR-based therapy is extremely promising, but costs over USD 1 million just to cover basic costs. It needs to be done at special facilities that aren’t available everywhere. Luckily, the things that need to be done do to evolve this therapy into the next generation are also what will decrease the costs, such as faster in vivo therapies, ones that can be done safely anywhere. “We are consciously trying to bend the cost curve down as quickly as possible to make sure that these therapies can be accessible to all who need them.” The groundbreaking CRIPSR/Cas9 findings are a two-sided discovery. The capability of making exact alterations to the human genome has revived discussions of moral concerns. The fact that the technique is so accessible has raised worry over potential misuse. The ethical implications of genome editing have occupied her mind ever since she started working on CRISPR, says Doudna. 58 NORDICLIFESCIENCE.ORG “Every powerful technology comes with tradeoffs, and CRISPR is certainly no exception. How we use it is up to us, and I’m really gratified that there has been so much discussion and thinking early on about how we track and regulate the use of gene editing. There have already been examples of people misusing the technology, but those have been minor compared to the vast amount of positive progress in medicine, agriculture, and as a basic research tool.” When Doudna’s Berkely colleague Professor Fyodor Urnov heard about the news at the beginning of October last year, the feeling of happiness and excitement was so overwhelming that he had the sensation of “levitating off his bed”, as well as a feeling of comfort, he said during a Zoom interview. “This was a rare moment in 2020 when the primary emotion was that the world is fair; exactly the right thing happened. The right discovery was honored and given to exactly the right people.” He highlights the fact that Doudna was a wellknown scientist before the Nobel Prize and before the CRIPSR discoveries. He talks about her as someone who is unafraid of difficult things, works with a tremendous discipline, pays attention to detail and devotes herself completely. “This wasn’t an accident that just landed in her lap. She is the top of her field. To use a sport analogy, we are talking about a world class athlete who has spent her life training for winning, and when a particular challenge came up, she showed us what she was capable of doing,” he says. A central driving force as a scientist is her curiosity. She sees puzzles and wants to solve them, Doudna says. “It’s the application of science that gets most people excited, but my work always starts with trying to answer fundamental questions and overcome barriers.” During our interview Urnov made an analogy to a story about Isaac Newton. When asked about how he discovered the laws of motion, he supposedly answered, “By thinking of them continuously.” “When you speak with Jennifer about the area of her research passion, the immediate thought that comes to mind is that she is of that Isaac Newton mindset. Someone who has been thinking continuously about it.” NLS