Gene Editing Pioneer David Liu: Fixing Baby’s Rare Disease Heralds New Medical Era

Prime editing-installed suppressor tRNAs for disease-agnostic genome editing

Precise genome-editing technologies such as base editing1,2 and prime editing3 can correct most pathogenic gene variants, but their widespread clinical application is impeded by the need to develop new therapeutic agents for each mutation. For diseases that are caused by premature stop codons, suppressor tRNAs (sup-tRNAs) offer a more general strategy. Existing approaches to use sup-tRNAs therapeutically, however, require lifelong administration…

Gene Editing Pioneer David Liu: Fixing Baby’s Rare Disease Heralds New Medical Era

The inventor of base and prime editing shares insights on medicine's efforts to make serious genetic diseases treatable or preventable, with recent clinical milestones.

What Could Go Right? - The Baby Who Got His Own Cure

This is our weekly newsletter, What Could Go Right? Sign up here to receive it in your inbox every Thursday at 5am ET. You can read past issues here. The Baby Who Got His Own Cure It was 2024, and a baby named KJ had just been born. He had his mother’s bright blue eyes, just like his three older siblings. Within days, however, it was clear something was seriously wrong. Little KJ was diagnosed with CPS1 deficiency, a rare liver condition in whic…

David Liu’s lab devises one-size-fits-many prime editing, but obstacles to broad use in humans persist

Researchers led by Broad Institute gene editing star David Liu have come up with a new use for prime editing that can tackle a bucket of diseases with the same root cause, which they say opens up a more straightforward path to address dozens or even hundreds of genetic diseases.