Gene therapy for multiple endocrine neoplasia type 1 (MEN1)

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The basic concept of gene therapy is simple, and this is to introduce a normal gene into a cell that has a defective gene, and the product of the normal gene should cure or slow down the progression of the disease. I propose to use this approach to treat endocrine tumours in the disorder of Multiple Endocrine Neoplasia type 1 (MEN1) that results from a single gene defect. Medical and surgical treatments are frequently unsuccessful in this disorder which is inherited and starts at an early age. I propose to use gene therapy to treat these tumours. Professor Thakker‘s group, at The University of Oxford, have identified the MEN1 gene and established a mouse model that develops the same tumours that are found in MEN1 patients. I now propose to attach the MEN1 gene to a modified virus (i.e. one with its harmful genes removed), to develop a gene therapy for MEN1 patients. I will test the effectiveness and safety of this MEN1 replacement virus in tumour cells and then use it to treat tumours in the mouse model. The results, if successful, will provide an important new treatment for MEN1 patients.