BioengineeringbioRxiv
Cure of congenital purpura fulminans via expression of engineered protein C through neonatal genome editing in mice
T. Togashi, N. Baatartsogt, Y. Nagao…·T. Ohmori
The paper demonstrates that ectopic expression of an engineered protein C via neonatal genome editing cures congenital protein C deficiency in mice.
DOI 10.1161/atvbaha.123.319460