C11950: Novel Inbred Mouse Model of Hemophilia ANovelty:
This invention is a transgenic inbred mouse model of Hemophilia A.
Value Proposition:
Currently available natural canine models of Hemophilia A are ineffective in designing gene therapy for the disease. There is a need for a mouse model to study the Factor VIII gene (Factor VIII deficiency causes Hemophilia A). This technology is a transgenic mouse model of Hemophilia A that is inbred with the commonly used C57B/6 mouse strain.
Advantages of this invention include:
• Easy for comparison with control-condition C57B/6 mice
• Smaller and easier to use than other larger animal models
• Apt for studying and designing tissue specific gene correction/therapy without immunologic rejection
• Suitable for testing and optimizing various gene delivery systems
Technical Details:
Johns Hopkins researchers have developed a strain of genetically altered mouse model of Hemophilia A. A neo cassette (reporting gene) was placed in exon 16 of Factor VIII gene in a mouse embryo and bred with C57B/6. The neo insertion knocked out Factor VIII, and produced a mouse model of hemophilia A.
Looking for Partners:
To commercialize this mouse model as a tool for Hemophilia A research and gene therapy.
Stage of Development:
Fully developed
Data Availability:
Tangible material
Publications/Associated Cases:
Nat Genet. 1995 May;10(1):119-21.