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American biologist Professor David Liu of the Broad Institute of MIT and Harvard has been honored with the prestigious $3 million Breakthrough Prize in Life Sciences. Liu was recognized for his pioneering contributions to the field of gene editing, particularly through the development of two groundbreaking technologies: base editing and prime editing. These innovations have opened new frontiers in the ability to precisely correct genetic mutations, offering hope for treating previously untreatable diseases.

Life-Saving Technology: A Teenager’s Battle with Leukemia

Liu’s technologies have already demonstrated their life-saving potential. In 2022, his base editing technique played a crucial role in the treatment of 13-year-old Alyssa Tapley at Great Ormond Street Hospital in London. After chemotherapy and a bone marrow transplant failed, doctors used base editing to modify her immune cells, enabling them to target and destroy her aggressive leukemia. Over two years later, Alyssa remains in full remission, a testament to the promise of this revolutionary technology.

Liu’s innovation represents a significant leap forward in precision medicine, providing an effective treatment for conditions that were once considered nearly impossible to treat.

A New Era of Gene Editing

Base editing, which allows scientists to correct single-letter mutations in the DNA, addresses a large portion of genetic diseases caused by these small errors. Prime editing, Liu’s more advanced technology, takes this further, functioning like a “search-and-replace” tool for DNA. It enables scientists to rewrite entire sections of the genetic code, offering the potential to correct almost all known genetic mutations responsible for inherited diseases.

These tools offer immense promise for curing a variety of genetic disorders. Over a dozen clinical trials are already underway to test the effectiveness of these technologies for conditions such as leukemia, sickle cell disease, beta-thalassemia, and high cholesterol. However, a significant challenge remains: the ability to edit cells inside the human body. Most clinical successes to date have involved editing cells outside the body, which are then reinfused into patients.

From Backyard Experiments to Global Impact

Liu’s journey into the world of science began in his childhood, where he spent hours experimenting with bugs in his California backyard. His academic path led him to Harvard, where he worked alongside Nobel laureate E.J. Corey. Liu’s creative spirit flourished as he combined scientific inquiry with innovation, designing slow-flying model planes, building LEGO-powered catapults, and even mastering blackjack—skills that eventually led to being banned from Las Vegas casinos.

Since the COVID-19 pandemic, Liu has made a significant commitment to supporting his research team. He has donated his entire post-tax salary each year to the students and staff in his lab, recognizing their hard work and dedication. Additionally, Liu founded a scientific foundation to promote biomedical research, which has gained further momentum with the support of his Breakthrough Prize.

Warning About Cuts to Scientific Funding

Despite the tremendous progress represented by his work, Liu has expressed serious concerns about the future of science, particularly in the United States. The threat of budget cuts and layoffs in scientific institutions has led Liu to warn against the long-term consequences of such actions. He likens these cuts to “burning your seed corn” and argues that reducing investment in science undermines efforts to improve the future of humanity.

Liu believes that science should focus on making life better for future generations, especially in terms of safety and health. He emphasized, “What could be more human than using all of our knowledge and effort to make our children’s lives safer and better than our own?”

The Future of Genetic Medicine

Prof. Liu’s work not only marks a significant step forward in the realm of genetic medicine but also serves as a reminder of the profound impact science can have on human lives. His innovations in gene-editing technologies hold the potential to transform the treatment of genetic diseases, offering new hope for millions worldwide.

As the scientific community looks toward the future, Liu’s achievements highlight the importance of continued investment in research and development. The field of gene editing is entering a new era, with the potential to revolutionize how we treat genetic disorders. However, this progress relies on strong support for science and technology—something Liu advocates for passionately.