Saving one life at a time: Gene-editing in clinical use Baby KJ was born in August 2024 with an ultra-rare genetic condition – carbamoyl-phosphate synthetase (CPS1) deficiency. The CPS1 enzyme, in the liver, converts ammonia (from broken down proteins) to carbamoyl phosphate. Mutations in the CPS1 gene cause carbamoyl phosphate synthetase I deficiency. CPS1 deficiency affects 1 in 1,300,000 persons. Defects in […]

There’s a very specific kind of silence in a molecular biology lab; the silence right after you finish an extraction experiment and you’re waiting to know whether it actually worked. It’s not peaceful. It’s not calm. It’s the kind of silence where even the centrifuge seems to hold its breath.  Everything before this moment feels loud: the vortex, the pipette clicks, the mental arguments with buffers, the “why is this viscous […]