FigureĀ 1.
RNA therapeutics for hemophilia. Sites of action of RNA therapeutics in the coagulation cascade. CRISPR/Cas9 gene editing and mRNA therapeutics target the genes encoding FVIII and FIX in the intrinsic coagulation pathway. The siRNAs target natural coagulation inhibitors of factors in the common coagulation pathway (FIIa, FVa, and FXa), including AT (siRNA AT), siRNA PS, and siRNA HCII.