FOr in the past five years, David Liu – Professor at the Broad Institute in MIT and Harvard, a biomedical research center in Massachusetts – has marked Thanksgiving by handing over his entire annual salary, after taking care of taxes, staff and students from his laboratory.
It started as the pandemic broke and that Liu heard that students who wanted to cycle instead of taking public transport could not afford bikes. Given how hard they worked and how paid they were, Liu intervened. He could not unilaterally increase their income, so sent them Amazon Egift cards. It also encountered problems. “Everyone thought they were scammed,” he recalls. And so he went to writing checks.
As a co-founder of several companies, Liu can reach both ends without her Harvard salary and has created a charity to continue scientific research. Its chests should considerably increase now that Liu received the breakthrough of $ 3 million for the life sciences, which was presented on Saturday at the annual award ceremony in Los Angeles.
The revolutionary prices, described by their founders of Silicon Valley as the Oscars of Sciences, are awarded each year to the scientists and mathematicians chosen by the previous winners. This year, two other prizes in the life sciences have been awarded for historical research on multiple sclerosis and the agonists of GLP-1, better known as “lean kicks”.
The other winners of the evening were Dennis Gaitsgory, a mathematician in Bonn, for his work on the Langlands program, an ambitious effort to unify the disparate concepts in mathematics, and more than 13,000 CERN researchers to test the modern theory of particle physics.
Liu was chosen to invent two exceptionally precise gene editing tools, namely the basic edition and the first -rate edition. The basic edition was first used in a patient in Great Ormond Street in London, where he saved the life of a British teenager in leukemia.
Scientists have been working on genes for more than a decade. Progress, they hope, will lead to therapies that correct the mutations responsible for thousands of genetic diseases. But the first generation of gene editing tools had a limited success: they were good at deactivating defective genes, but not to correct them.
The basic edition allows scientists to make changes to unique letters from the genetic code, while the main edition has been compared to the research and replacement function in text processing, giving researchers the power to rewrite whole DNA stretching. Together, they have enormous potential. “The vast majority of known pathogenic mutations can now be corrected using the main edition or the basic edition,” explains Liu.
Liu grew up in Riverside, California, and traces her interest in science to play with bugs in her backyard. He went to Harvard and worked with EJ Corey, a Nobel Prize winner considered one of the greatest chemists in our time. “It was the beginning of what turned into a life for life of experimental molecular science,” explains Liu. “He encouraged me to follow my passions and my curiosity.”
His curiosity was not limited to chemistry. Liu read that radio-controlled airplane fans wanted an airplane that was flying slowly to pilot a room. After working on the equations, he built the WISP, a six -gram carbon fiber plane that zoomed in one mile per hour. Another project merged the Lego bricks with the heat sensor of a burglar alarm to produce the “Mousapult”, a device that detected cats and launched toy mice in their direction.
Video games also presented heavily. In the early 1990s, Liu dragged with Andy Gavin and Jason Rubin, the students behind the Naughty Dog game developer. He tested games and was an actor of occasional voice. A performance has done Warrior For the 3DO game machine. “I said something like …” He takes a break to adopt a mocking tone “…” my dead grandfather fights better than you “.
A more risky hobbin took root while Liu was at the hospital recovering from an operation. He wanted to beat the blackjack and write a simulator to understand mathematics. Before a long 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 prohibited from all the large MGM casinos and, to use the game euphemism, “behind” twice to read the laws on the intrusion of Nevada.
Later, as a professor at Harvard, a group of students convinced Liu to lead a course on the counting of the cards. “The best decision I made about this team was that no member put their own money and that no member took their own money. “It was the pleasure of learning something really difficult.”
In the laboratory, Liu was trying to solve a very different problem. Gene edition At the time, could deactivate genes, but not rewrite the letters from the DNA code. But disabling genes would never be enough to treat genetic diseases. “They must be treated by repairing the gene,” he says.
THE first breakthrough In 2016 came when the Liu team described the basic edition, a way to correct mutations to a single letter which represent almost a third of genetic diseases. The procedure used Crispr Guide to molecules to find the defective code and an enzyme to modify the aberrant letter. Waseem Qasim, pediatric immunologist at Great Ormond Street Hospital, remembers reading the newspaper during breakfast the day after its publication. “My children were relatively small at the time. I spit on my cornflakes and I said: Look at this, guys, science fiction! ”
A tracking document In 2019, described the Prime edition, a less effective but more powerful technique which can in principle repair almost all pathogenic mutations.
The advantages of the basic edition became clear in 2022 when the Qasim team became the first in the world to use the procedure on a patient. Alyssa TEPLEYA 13 -year -old child of Leicester, had lacked options after chemotherapy and a bone marrow transplant had not treated his leukemia. Cancer has affected its T cells, a group of immune cells that normally fight in infections.
Doctors collected T cells with a healthy donor and modified the genetic code so that when they were infused in Alyssa, they would seek and attack its cancer cells. The treatment worked: more than two years later, Alyssa remains in complete remission.
More than a dozen clinical trials are now underway to test the basic edition and the main edition. Positive results have already been reported for leukemia, sickle cell,, Beta-Thallasemia And high cholesterol. But major obstacles remain. Although Alyssa’s treatment involved cell editing outside the body and sending them, most diseases require mutations to be fixed inside the patient. This is something that scientists have not yet cracked.
This is not the only problem. The Qasim team treats more patients in a test, but at the end of the test, there may be no one to finance future treatments. “We are going to finish with treatments that work, but that no one wants to pay.”
Liu is optimistic that researchers can find ways to deliver therapies and reduce costs, but he has major concerns about the future of science, especially in the United States. He thinks that the recent wave of layoffs and financing cuts is an existential threat to the next decade or two of progress that will have ramifications in the world.
“For me, reducing funding and people in science in the United States is like burning seed corn. He doesn’t even eat the corn of your seeds. He is only destroying it, ”he says. “What could be more human than wanting to use all our knowledge, all our efforts, all our resources, to try to make the lives of our children safer and better than our own life? A large part of this aspiration requires, and is indeed motivated by science. ”