The scientist rewriting DNA, and the way forward for drugs

A revolution is underway in gene modifying—and at its forefront is David Liu, an American molecular biologist whose pioneering work is rewriting the constructing blocks of life with unprecedented precision.

A professor on the Broad Institute of MIT and Harvard, Liu was awarded a Breakthrough Prize in Life Sciences on Saturday for creating two transformative applied sciences: one already enhancing the lives of sufferers with extreme genetic ailments, the opposite poised to reshape drugs within the years forward.

He spoke with AFP forward of the Los Angeles ceremony for the distinguished Silicon Valley-founded award.

He’ll obtain $3 million for his work on “base modifying” and “prime modifying,” and plans to donate most of it to assist his charitable basis.

“The power to alter a DNA sequence of our selecting into a brand new sequence of our selecting is a basically very highly effective functionality,” the 51-year-old stated, foreseeing makes use of not simply in human drugs however areas like creating extra nutritious or disease-resistant crops.

Correcting the code

DNA is made up of 4 chemical “letters”—the nucleotide bases A, G, T and C. Mutations on this sequence trigger 1000’s of human ailments, but till not too long ago, gene modifying might solely repair a restricted variety of them.

Even CRISPR-Cas9, the groundbreaking know-how that earned a Nobel Prize in 2020, has main limitations.

It cuts each strands of the DNA helix, making it most helpful to disrupt quite than right genes, whereas the method can introduce new errors.

“Having the ability to use genome modifying to deal with genetic ailments requires, normally, methods to right a DNA misspelling, not merely to disrupt a gene,” Liu stated.

That perception led his lab to develop base modifying, which makes use of the Cas9 protein—disabled so it may well not reduce each DNA strands—to discover a goal DNA sequence and one other enzyme to transform one letter to a different—for instance, C to T or G to A.

Reversing the change—from T to C or A to G—was harder. Liu’s group overcame the problem by engineering completely new enzymes.

These base editors can now right about 30% of the mutations that trigger genetic ailments. The know-how is already in a minimum of 14 medical trials.

In one in every of them, Beam Therapeutics—which Liu co-founded—introduced it had handled sufferers of AATD, a uncommon genetic dysfunction affecting the lungs and liver, with a single drug infusion.

Whereas conventional gene therapies usually disrupt defective genes or work round them, base modifying repairs the mutation itself.

“This was the primary time that people have corrected a mutation that causes a genetic illness in a affected person,” Liu stated.

Cystic fibrosis hope

Base modifying, shortly dubbed “CRISPR 2.0,” cannot repair each mutation.

About 70% of the roughly 100,000 recognized disease-causing mutations stay out of its attain, together with these brought on by lacking or further letters.

To increase the toolkit, Liu’s lab launched prime modifying in 2019—a way able to changing complete sections of defective DNA with corrected sequences.

If CRISPR is like scissors that reduce DNA, and base editors are like utilizing a pencil to right particular person letters, then prime modifying is the equal of a phrase processor’s “discover and change” operate.

Creating this device required a collection of breakthroughs Liu’s group describes as “small miracles.” The result’s, he stated, “probably the most versatile method we all know of to edit the human genome.”

Among the many targets Liu and his group have already pursued with prime modifying: cystic fibrosis, a standard genetic illness normally brought on by three lacking DNA letters that causes thick mucus buildup within the lungs and digestive system.

Liu’s lab has made a lot of its work freely accessible, sharing DNA blueprints by a nonprofit library utilized by tens of 1000’s of labs worldwide.

“The science we create—which is in the end funded by society, by governments and donors—in the end goes again to learn society.”

This yr’s Breakthrough Prize awards come at a fraught second for U.S. science, as President Donald Trump’s authorities strips funding for establishments just like the Nationwide Institutes of Well being (NIH).

“The NIH is a treasure, not only for this nation however for the world,” stated Liu. “Making an attempt to dismantle the center of what helps science on this nation is like burning your seed corn.”

© 2025 AFP