This is what we are talking about when we say NIH funding saves lives. Specifically these researchers stepped in created a therapy that may well save this child's life. It is amazing. This is what is at stake. If NIH goes away, we lose all of this, for at least a generation.
Ted Price
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a ureacycle disorder. The affected infant was treated at approximately 7 and 8 months of age. Read the Brief Report: nej.md/42XF03F
#ASGCT2025 #MedSky @asgct.bsky.social
Base editors can correct disease-causing genetic variants. After a neonate had received
a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with
an estimated 50% mortality ...
