We propose the creation of ionizable lipid nanoparticles (LNPs) containing mRNAs that encode the coagulation factor VIII protein.
LNPs administered into the bloodstream travel to the liver and target LSECs with the help of surface proteins.
LNPs administered into the bloodstream travel to the liver and target LSECs with the help of surface sugars.
Once inside the cells, the LNPs will release the mRNAs, leading to the production of coagulation factor VIII.
This approach enables patients to produce their own coagulation factor VIII, eliminating the need for frequent injections of plasma-derived or recombinant proteins.
