The huFVIII-R593C transgene was designed with the murine albumin (Alb) promoter driving expression of a mutated human coagulation factor VIII (F8) cDNA. Mice carrying this transgene were bred to mice deficient in endogenous F8. F8 is a coagulation factor, and mutations of this gene have been linked to hemophilia A. As such, the donating investigator reports that these mice will die due to blood loss from a tail clip (see additional note below). These double mutant mice are tolerant of human F8 (hF8) injection, and have no F8 activity. They produce very little inhibitory antibodies and lack F8 responsive T cells or B cells after treatment with human F8. When treated with a hF8 expression plasmid driven by a liver specific human &alpha;1 anti trypsin promoter and an attB phiC31 integrase site, in conjunction with phiC31 integrase treatment, this hF8 integrates into the host genome at specific "pseudo-attP sites" and results in long-term gene therapy. This strain may be useful for studying hF8 gene therapy treatments for Hemophilia A patients. Additional note on tail clipping: The donating investigator reports that these mice will die due to blood loss from a tail clip. The donating investigator suggests no tail clipping be performed on mice younger than 4 weeks of age, and tail clipping with cauterization may be used for mice older than 4 weeks of age.

These mice contain a huFVIII-R593C transgene, designed with the murine albumin (Alb) promoter driving expression of an altered human coagulation factor VIII (F8) cDNA harboring a mutation that is frequently observed in patients with mild hemophilia A. These mice also carry a knock-out of the F8 gene and are deficient for endogenous F8 protein.

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STOCK F8<tm1Kaz> Tg(Alb-F8*R593C)T4Mcal/J Frozen Embryo