How Gene Editing Is Changing Genetic Disorder Treatment

Harvard chemist David Liu has transformed medicine by inventing gene editing tools that correct genetic mutations without cutting the DNA double helix.

Around 2011 he and graduate student Kevin Esvelt developed PACE, or phage assisted continuous evolution, which harnesses fast reproducing bacteriophages to run 200 generations of protein evolution in about eight days rather than a full year.

Building on that work, his lab created base editing in 2016 with Alexis Komor and later prime editing with Andrew Anzalone, techniques that rewrite individual genetic letters to fix mutations behind diseases like sickle cell anemia.

At least 23 clinical trials now rely on his methods to treat conditions including lung cancer and metabolic disorders.

One patient, a 12 year old English girl named Alyssa Tapley, saw her leukemia driven back in 2022 when her immune cells were edited after conventional treatment failed, and she remains cancer free.

Liu stresses that such breakthroughs depend on sustained funding for curious young researchers, warning that future cures for diseases like progeria hinge on continued investment in basic science.

The gene rewriting tools David Liu invented, which fix mutations without cutting the DNA double helix, now run at least 23 clinical trials, all eight with reported results benefiting patients a BioCentury interview. Nothing is approved yet: the only cleared gene therapies, for sickle cell and beta thalassemia, still work by cutting DNA rather than rewriting letters Children's Hospital of Philadelphia. The sharpest evidence came in February 2025, when an infant got the first custom base editing infusion for a urea cycle disorder; a year on he walks and talks, though doctors call it treatment, not cure. Tiny patient pools deter drugmakers; Prime Medicine now asks the FDA to clear a prime edited therapy on data from just 2 patients.