David Liu, pioneer of base editing and prime editing wins major science prize

Professor David Liu, the developer of base editing and prime editing, has won the Breakthrough Prize in Life Sciences, the largest annual monetary award in science.

The Breakthrough Prize in Life Sciences seeks to recognise people who have conducted groundbreaking research. Professor Liu from the Broad Institute of MIT and Harvard, in Cambridge, Massachusetts, has pioneered novel approaches to advance the field of genome editing. Two such approaches are 'base editing' (see BioNews 848 and 924) and 'prime editing' (see BioNews 1021 and 1243), which enable precision genome editing without double-stranded breaks. This can avoid some of the off-target effects, and unwanted on-target effects, that can otherwise occur in CRISPR genome editing.

'The real heroes behind our work are the incredibly talented graduate students, postdocs, and collaborators who worked tirelessly to develop these technologies in ways that would allow them to benefit society,' said Professor Liu. 'Without their dedication, this work would not be possible.'

Base editing was developed in 2016 with the a view to treating diseases associated with single genetic mutations. Some genetic diseases, including sickle-cell anaemia and certain cancers, are caused by a single base pair change in a gene. In 2022, base editing was used for the first time to treat a teenager with T-cell leukaemia at the Great Ormond Street hospital in London (see BioNews 1172).

The human genome consists of base-pairs – adenine always pairs with thymidine, and cytosine always pairs with guanine. Base editing allows scientists to edit single bases within DNA, for example converting a cytosine-guanine base pair to an adenine-thymidine base pair using a cytosine base editor, or converting an adenine-thymidine base pair to a guanine-cytosine base pair using an adenine base editor.

Prime editing was developed in 2019 in order to be able to edit more than one base at a time. In more traditional CRISPR/Cas9 genome editing, the Cas9 enzyme cuts both strands of DNA, which is useful for disrupting genes but less useful for correcting them. Prime editing uses a modified Cas9 that has a reverse transcriptase enzyme attached, in order to cut just one strand of double-stranded DNA, and introduce new DNA from an RNA template included in the prime editor. The US FDA approved clinical trials using prime editing in 2024.

'It has been my privilege to support David's work over the years,' said Dr Richard Merkin, CEO and founder of Heritage Provider Network. 'He has a clear vision for his work and is committed to seeing it improve human health. I'm delighted to see him recognised in this way.'

Having corresponded with Professor Francis Crick (one of the discoverers of the structure of DNA, see BioNews 269 and 700) when he was a teenager, Professor Liu began his academic career in earnest as an undergraduate student at Harvard. After achieving his PhD at the University of California, Berkeley, he returned to Harvard became a professor there, before going on to become a core member of the Broad Institute.

In 2017, Professor Liu was named one of Nature's top ten most influential researchers in the world. Professor Liu, and his graduate and postdoctoral students, intend to continue refining base editing and prime editing in order to better deliver therapeutics to patients.