David Liu integrates chemistry and evolution to advance our understanding of biology and to enable new therapeutics. His research team’s work includes: the engineering and delivery of genome editing proteins, such as base editors, to study and treat genetic diseases; the use of phage-assisted continuous evolution (PACE) for proteins with novel therapeutic potential; and the use of DNA-templated organic synthesis to discover bioactive synthetic small molecules and synthetic polymers. These technologies – base editing, PACE, and DNA-templated synthesis – were pioneered in Liu’s laboratory.
A team led by HHMI Investigator David Liu used prime editing — a precise and versatile form of gene editing — to correct the root cause of a rare and severe neurological disorder in a laboratory model. The new research suggests a potential path to treating the disease that currently has no cure or effective treatment.   A gene editing tool based on a bacterial toxin can make precise changes to mitochondrial DNA inside cells. CRISPR has sparked a renaissance in genome editing. Now, next-generation CRISPR technologies let scientists modify the genome more efficiently and precisely than before. Such tools could one day serve as therapeutics, but many challenges remain. A single treatment of a genome editing agent partially preserved hearing in mice with genetic deafness. The work could one day help scientists treat certain forms of genetic hearing loss in humans. A new type of DNA editing enzyme, developed in HHMI Investigator David Liu’s lab, lets scientists directly and permanently change single base pairs of DNA from A•T to G•C. The process could one day enable precise DNA surgery to correct mutations that cause human diseases.