Innovation Biologist who saved the British young man’s life won the $ 3 million breakthrough award | Gene regulation

FOr in the last five years, David Liu, a wide professor of MIT and Harvard, a biomedical research facility in Massachusetts, marked the Taxes and students in the Laboratory by delivering taxes and annual salary to students.

When my pande was broken, Liu heard that students who want to bicycle instead of buying public transport could not get bicycles. Given how hard they worked and how little they were paid, Liu came in. He could not raise his revenues unilaterally, so he sent E -mail to the Amazon Egift cards. However, this also faced problems. “Everyone thought he was defrauded,” he remembers. And so he went into writing checks.

Liu, the founding partner of several companies, can end without a Harvard salary and formed a philanthropist basis for further advanced scientific research. The serers are significantly swelling because Liu received the $ 3 million Life Sciences Award on Saturday at the annual award ceremony in Los Angeles.

The breakthrough awards, defined by the founders of the Silicon Valley as Science Oscars, are given to scientists and mathematicians chosen by previous winning committees. This year, two lifelore awards, known as “Silka Jablar için for the turning point research on multiple sclerosis and GLP-1 agonists, were awarded.

The other winners of the night, Dennis Gaitsgory, a mathematician in Bonn, were to test the theory of modern particle physics of more than 13,000 researchers in CERN, an ambitious effort to unite different concepts in mathematics.

Liu was chosen to invent two extraordinary sensitive gene editing tools, basic arrangement and prime arrangement. The basic arrangement was first used in a patient on Great Ormond Street in London, where a British young man saved his life with leukemia.

Scientists have worked on gene arrangement for more than ten years. Progress will lead to therapeutics that correct mutations responsible for thousands of genetic diseases. However, the first generation gene regulation tools had a limited success: it was good to disable faulty genes, but they were not in correcting them.

While the basic arrangement allows scientists to make changes to the single letters of genetic code, the main regulation is compared with the search and replacement function in a word processor and gives researchers the power to rewrite the whole of DNA. They have a tremendous potential together. Li The majority of known pathogenic mutations can now be corrected using the main arrangement or basic arrangement, Li says Liu.

Liu grew up in Riverside, California and watching his interest in science with insects in his backyard. He went to Harvard and worked with Nobel Prize -winning Ej Corey and was considered one of the greatest chemists of our time. “This was the beginning of what turned into a lifetime of experience in experimental molecular science, Li says Liu. “He encouraged me to follow my passions and curiosity.”

His curiosity was not limited to chemistry. Liu read that radio -controlled aircraft enthusiasts wanted a slow flying plane around a room. After running the equations, he built WISP, a six -gram carbon fiber plane, a slow mile closer per hour. Another project combined LEGO bricks, cats perceiving and toy mice, a device that detects toy mice, a alarm of a thief to produce “mouseapult ..

Video games also took part intensively. In the early 1990s, Liu was attached to the students behind the game developer Naughty Dog, Andy Gavin and Jason Rubin. He tested the games and was occasionally a voice player. A performance did this Warman’s Way For 3do game machine. “I said something like this…” He pauses to adopt a cynical tone…… ‘My dead grandfather is fighting better than you’. ”

Liu gets rid of an operation and a more risky hobby rooted. He wanted to beat Blackjack and wrote a simulator to understand mathematics. Soon he worked on a series of card counting techniques and went to Las Vegas to test them. He did so well that he was banned from all MGM Grand casinos and twice to read the unauthorized entry laws to use the game covering.

Later, as a professor in Harvard, a group of students convinced Liu to teach Liu a lesson in the card count. “The best decision I made about this team was that no member did not put their own money and no member did not get their own money. Everything returned to the fund to fly to Las Vegas and pay for our hotels and meals,” he says. “Everything was about the fun of learning something really hard.”

Liu was trying to break a very different problem in the laboratory. At that time, the gene regulation can disable genes, but cannot rewrite the letters of the DNA code. However, deactivating genes is never enough to treat genetic diseases. “They need to be treated by fixing the gene, or he says.

. First breakthrough In 2016, when Liu’s team described the basic arrangement, a way of correcting one -letter mutations, which constitute approximately one -third of genetic diseases. The procedure used an enzyme to change CRISPR guide molecules and abnormal letters to find the wrong code. Waseem Qasim, a pediatric immunologist at Great Ormond Street Hospital, remembers that he read the article on breakfast after release. “My children were relatively small at that time. I spit my corn flakes and I said, here you are, science fiction!”

A follow -up article In 2019, he described the Prime Editing, a less efficient but more powerful technique that could repair the mutations that cause almost all the disease.

Benefits of the Basic Regulation was found to be the first person to use the procedure in a patient in the world in 2022. Alyssa Tapley, a 13 -year -old of Leicester, was exhausted after chemotherapy and could not treat a bone marrow transplant leukemia. Cancer affected T cells, a group of immune cells that normally fight infections.

The doctors gathered T cells from a healthy donor and changed the genetic code so that they would call and attack cancer cells when they were vaccinated in Alyssa. Treatment studied: Alyssa remains in full remission after more than two years.

More than a dozen clinical work continues to test the basic arrangement and prime regulation. Positive results have already been reported for leukemia, Sickle Cell Disease– Beta-Thallasa And high cholesterol. However, great obstacles remain. Although Alyssa’s treatment involves editing and sending cells outside the body, most diseases require mutations to be fixed in the patient. This is a trick that scientists have not yet cracked.

This is not the only problem. Qasim’s team treats more patients in an experiment, but at the end of the trial, there may be no one to finance future treatments. “We will result in working treatments, but no one wants to pay.”

Liu is optimistic that researchers can find ways to present therapies and reduce costs, but they have serious concerns about the future of science in the United States. He believes that the progress of recent fire and financing segments, which will have worldwide consequences for the next decade, pose an existential threat.

“In my opinion, it is like reducing financing and burning your seed corn in the United States. “What can be more human than using all our knowledge, all our efforts, all our resources to make our children’s lives safer and better than our own lives? A large part of this aspiration requires science and really directed.”