Cure of congenital purpura fulminans via expression of engineered protein C through neonatal genome editing in mice
T. Togashi, N. Baatartsogt, Y. Nagao, Y. Kashiwakura, M. Hayakawa, N. Kamoshita, T. Hiramoto, T. Fujiwara, E. Morishita, O. Nureki, T. Ohmori
The paper demonstrates that ectopic expression of an engineered protein C via neonatal genome editing cures congenital protein C deficiency in mice.
bioRxiv · Feb 26, 2023Bioengineering