A professor at the Broad Institute of MIT and Harvard, Liu was awarded a Breakthrough Prize in Life Sciences on Saturday for developing two transformative technologies: one already improving the lives of patients with severe genetic diseases, the other poised to reshape medicine in the years ahead.
He will receive $3 million for his work on “base editing” and “prime editing,“ and plans to donate most of it to support his charitable foundation.
Correcting the code
Even CRISPR-Cas9, the groundbreaking technology that earned a Nobel Prize in 2020, has major limitations.
“Being able to use genome editing to treat genetic diseases requires, in most cases, ways to correct a DNA misspelling, not simply to disrupt a gene,“ Liu said.
Reversing the change -- from T to C or A to G -- was tougher. Liu's team overcame the challenge by engineering entirely new enzymes.
In one of them, Beam Therapeutics -- which Liu co-founded -- announced it had treated patients of AATD, a rare genetic disorder affecting the lungs and liver, with a single drug infusion.
“This was the first time that humans have corrected a mutation that causes a genetic disease in a patient,“ Liu said.
Base editing, quickly dubbed “CRISPR 2.0,“ can’t fix every mutation.
To expand the toolkit, Liu's lab introduced prime editing in 2019 -- a method capable of replacing entire sections of faulty DNA with corrected sequences.
Creating this tool required a series of breakthroughs Liu’s team describes as “small miracles.” The result is, he said, “the most versatile way we know of to edit the human genome.”
Liu's lab has made much of its work freely accessible, sharing DNA blueprints through a nonprofit library used by tens of thousands of labs worldwide.
This year's Breakthrough Prize awards come at a fraught moment for US science, as President Donald Trump's government strips funding for institutions like the National Institutes of Health (NIH).
“The NIH is a treasure, not just for this country but for the world,“ said Liu. “Trying to dismantle the heart of what supports science in this country is like burning your seed corn.”
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