To express a recombinant protein in the milk of an animal, expression vectors containing a gene encoding the protein of interest are fused to milk-specific regulatory elements (such as casein, lactalbumin or lactoglobulin)

and introduced by microinjection of a one-cell embryo, or alternatively transfected into a cell line suitable for somatic cell nuclear transfer. The mammary-gland specific transgene is transmitted in a Mendelian fashion, following integration into the germline. If expressed, it becomes a dominant genetic characteristic that HER2 inhibitor will be predictably inherited by offspring of the animal, and the yield of transgenic protein in the milk is often high in the range of grammes per litre. Transgenic expression delivers the advantages of mammalian cells (such as sophisticated molecular refolding machinery and glycosylation), as well as the potential for flexibility of scale in production and relatively low costs. Pigs have proved to be the best animals to produce human coagulation proteins: the γ- carboxylation profile of factor IX from pigs is much better than that observed in sheep. There is no evidence of transmission of porcine pathogens to humans and, in particular, pigs are not susceptible

to BSE/vCJD. They also have a high reproductive rate and each sow can produce 200–300 L of milk per year, which is unusually rich in protein. Early experiments using transgenic factor IX in haemophilic dogs have already begun (WH Velander, personal communication). In conclusion, blood products made from porcine plasma had an important role in the treatment of haemophilia selleckchem for half a century from 1954 to 2004. A recombinant porcine factor VIII product is now at an advanced stage of clinical development. In the more distant future, the distinction between human and porcine products may become blurred through the production of hybrid human/porcine

selleck chemicals llc recombinant factor VIII, and even human factor VIII produced in transgenic pigs. I am grateful to Dr P. Robinson of Ipsen for discussions about the development of Hyate: C and providing the photographs which appear as Figs 2–4 in this article. I am also grateful to Sally Tranter and Graham Joint of Communigen for editorial assistance. The author stated that he had no interests which might be perceived as posing a conflict or bias. “
“Inhibitor development against exogenous factor VIII is a severe impairment of replacement therapy affecting 18% of Argentine patients with severe haemophilia A (HA). To study the molecular predisposition for inhibitor development, we genotyped 260 HA patients with and without inhibitors, countrywide. The inhibitor-positive population (19 transients, 15 low responders, LR and 70 high responders, HR) of 104 severe-HA patients showed 59 Inv22 (intron 22 inversions), 18 small ins/del-frameshifts, 12 gross deletions, 12 nonsense, one splicing defect and two missense, p.Arg531Pro and p.