The biologist whose innovation saved the life of British teenager has won a revolutionary prize from $ 3 million | Genetic editing

FOr over the past five years, David Liu – professor at the large institute of Mit and Harvard, a biomedical research structure in Massachusetts – has marched thanks by delivering all his annual salary, after taking care of the taxes, staff and students of his laboratory.

It started when the pandemic broke and Liu heard that the students who wanted to ride a bicycle instead of getting public transport could not afford the bicycles. Given how much they worked and how little they were paid, Liu intervened. He was unable to unilaterally lift their income, so he sent them an E -mail with the Amazon Egift cards. This also had problems, however. “Everyone thought they were scammed,” he recalls. And so it passed to the writing controls.

As co-founder of several companies, Liu can land the Lunario without his Harvard salary and has set up a charity base for further scientific research. His speakers are due to inflate considerably now that Liu has received the Breakthrough prize for the life sciences of $ 3 million, with which he was presented to him on Saturday at the annual award ceremony in Los Angeles.

The innovative prizes, described by their founders of Silicon Valley such as the Oscars of Science, are assigned every year to scientists and mathematicians chosen by the committees of previous winners. This year two other prizes of life sciences were awarded for the research of reference on multiple sclerosis and Glp-1 agonists, better known as “Jab Skinny”.

Other winners of the night were Dennis Gaitsgory, a mathematician in Bonn, for his work on the Langlands program, an ambitious effort to unify disparate concepts in mathematics and more than 13,000 researchers at CERN to test the modern theory of particle physics.

Liu was chosen to invent two exceptionally precise gene editing tools, that is to say the basic editing and the main modification. The basic assembly was used for the first time in a patient at Great Ormond Street in London, where it is Saved the life of a British teenager with leukemia.

Scientists have been working on gene editing for more than a decade. The progress, hope, will lead to therapies that correct mutations responsible for thousands of genetic diseases. But the first generation of gene editing tools had a limited success: they were good at disabled the defective genes, but not to correct them.

The basic editing allows scientists to make changes to the individual letters of the genetic code, while primary editing has been compared with the research and replacement function in a texture of texts, giving researchers the power to rewrite entire traits of DNA. Together, they have enormous potential. “The vast majority of pathogenic mutations known can now be correct using primary editing or basic editing,” says Liu.

Liu grew up in Riverside, California, and traces his interest in science to play with insects in his courtyard. He went to Harvard and worked with Ej Corey, a Nobel Prize considered one of the greatest chemists of our time. “This was the beginning of what was transformed into a permanent love for experimental molecular science,” says Liu. “He encouraged me to follow my passions and my curiosity.”

His curiosity was not limited to chemistry. Liu read that fans of planes on radio-controlled wanted a plane that flown slowly enough to pilot in a room. After working at the equations, he built the WISP, a six -gram carbon fiber plane that enlarged a pleasant mile per hour. Another project joined LEGO bricks with the heat sensor from a thief alarm to produce “mouseapult”, a device that detected cats and mice toys in their direction.

Video games also had heavily. At the beginning of the 90s, Liu came out with Andy Gavin and Jason Rubin, the students behind the Naughty Dog games developer. He tested the games and was an occasional voice actor. A performance has arrived Way of the Warrior For the 3Do games machine. “I said something like …” he does a break to adopt a mocking tone “… my dead grandfather fights better than you”. “

A more risky hobby took root while Liu was in the hospital to recover from an operation. He wanted to beat Blackjack and wrote a simulator to understand mathematics. In a short time, he had developed a series of cards counting techniques and went to Las Vegas to test them. He did so well that he was banned from all the big MGM casinos and, to use the mild game, “Back-Ro-Camera” twice to read the laws on the transgression of Nevada.

Later, as a professor in Harvard, a group of students convinced Liu to keep a lesson on the counting of the cards. “The best decision I made on that team was that no member put his money and no member took his own money. Everything returned to the bottom for us to fly to Las Vegas and pay for our hotel and meals,” he says. “It concerns the fun of learning something really difficult.”

In the laboratory, Liu was trying to break a very different problem. Genetic editing At the time he could disable genes, but not to rewrite the letters of the DNA code. But disabled genes would never be enough to treat genetic diseases. “They must be treated by fixing the gene,” he says.

THE First turning point It arrived in 2016 when Liu’s team described basic editing, a way to correct mutations in single letters that represent almost a third of genetic diseases. The procedure used CRISPR Guide the molecules to find the defective code and an enzyme to change the aberrant letter. Waseem Qasim, a pediatric immunologist at the Great Ormond Street Hospital, remembers having read the newspaper for breakfast the day after it was published. “My children were relatively young at the moment. I spit on my cornflakes and I said, I look at this, guys, science fiction!”

A follow-up card In 2019 he described privileged assembly, a less efficient but more powerful technique that in principle can repair almost all mutations that cause the disease.

The advantages of the basic modification became clear in 2022 when the Qasim team became the first in the world to use the procedure on a patient. Alyssa TapleyA 13 -year -old from Leicester had exhausted the options after chemotherapy and a bone marrow transplant had not been able to treat his leukemia. Cancer has influenced its T cells, a group of immune cells that normally fight infections.

The doctors collected T cells from a healthy donor and modified the genetic code so that if infused in Alyssa they would have sought and attacked its cancer cells. The treatment worked: more than two years later, Alyssa remains in complete remission.

More than a dozen clinical studies are now underway to test basic editing and primary assembly. Positive results have already been reported for leukemia, falciform anemia, Beta-talasaemia AND high cholesterol. But more obstacles remain. While the treatment of Alyssa involved the modification of the cells outside the body and the sending of them, most of the diseases require that the mutations were fixed within the patient. This is a trick that scientists have yet to break.

It is not the only problem. The Qasim team is dealing with more patients in a study, but when experimenting it ends, there may be nobody to finance future treatments. “We will end the treatments that work, but that nobody wants to pay.”

Liu is optimistic that researchers can find a way to provide therapies and reduce costs, but has serious concerns about the future of science, in particular in the United States. He believes that the recent wave of fires and financing cuts represents an existential threat for the next decade of progress that will have consequences all over the world.

“For me, cutting funding and people from science in the United States is like burning seed corn. He is not even eating corn for seeds. He is only destroying it,” he says. “What can be more human than wanting to use all our knowledge, all our efforts, all our resources, to try to make our children life safer and better than our life? A huge part of that aspiration requires and is really guided by science.”