Description:
Current State of the Art:
Studies in angiogenesis link new blood vessel formation to both wound healing and pathologies in the human body. Angiogenesis can be stimulated during regeneration of diseased or damaged tissues as well as during tumor growth and metastasis, in order to gain access to oxygen and nutrients necessary for survival. Gene therapy is a promising field to combat human disease by integrating new healthy gene products into a diseased or damaged state to influence recovery or impede disease progression.
Gene therapies targeted to new blood vessels would improve therapeutic applications for cancer, wound healing, diabetic retinopathy, rheumatoid arthritis and cardiovascular/arterial disease.
The Global gene therapy market is still in the experimental stages with only a few products in Phase II/III clinical trials. One gene therapy drug, Gendicine, has been approved and is marketed in China. Overall the industry is predicted to reach $480 million by 2015.
Disadvantages with the Current Art:
Companies moving to develop gene therapy technologies would benefit from a built-in modifier or a controlled, targeted aspect of the delivery of the therapeutic gene product. Otherwise irrevocable genetic changes made to the patient’s cells can have detrimental effects.
Advantages of Invention:
The inventors have engineered an optimized Alk1 promoter fragment which can be used in a variety of constructs including but not limited to an adenoviral vector. Endothelial cells containing therapeutic genes with this promoter will only express the therapeutic product in newly formed or remodeling arteries.
The inventors have developed a mouse model system that can be used to identify drugs that would be useful in either inhibiting or promoting new blood vessel formation.
Patent Status: US8431392
Inventor: Tsugio Seki, PhD
Case Number: GHSU 2009-024