Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
- 1 May 1995
- journal article
- letter
- Published by Springer Nature in Nature Genetics
- Vol. 10 (1) , 119-121
- https://doi.org/10.1038/ng0595-119
Abstract
Haemophilia A is a classic X-linked disease which affects 1 in 5–10,000 males in all populations1 and is caused by defects in coagulation factor VIII2,3. Roughly 60% of patients have severe disease with factor VIII activity 2. Natural canine models of factor VIII and factor IX deficiency have been available for many years4–5, and gene therapy attempts on these dogs have met with partial success6–8. However, a small animal model of the disease is desirable for studies of factor VIII function and gene therapy. Using gene targeting, we have made a mouse with severe factorVIII deficiency.Keywords
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