Although the primary goal of creating this mixed mouse model was to explore gene therapy for SPCD, recent studies by Magisetty et al. and Jiang M et al. have [15,16] demonstrated that a selective inhibition of APC’s anticoagulant activity, without affecting its cytoprotective signaling, reduced the severity of hemophilic arthropathy in FVIII-deficient mice. Here, F8 is linked to systemic primary carnitine deficiency disease.