Gene therapy for multiple endocrine neoplasia type 1 (MEN1)

基因治疗的基本概念很简单，就是将一个正常的基因引入到一个有缺陷基因的细胞中，正常基因的产物应该可以治愈或减缓疾病的进展。我建议用这种方法治疗由单基因缺陷引起的1型多发性内分泌瘤（MEN1）紊乱中的内分泌肿瘤。药物和手术治疗往往不成功，这种疾病是遗传的，在早期就开始了。我建议用基因疗法来治疗这些肿瘤。牛津大学Thakker教授的研究小组已经确定了MEN1基因，并建立了一个小鼠模型，该模型会产生与MEN1患者相同的肿瘤。我现在建议将MEN1基因附着在一种修饰过的病毒上（即去除有害基因的病毒），以开发一种针对MEN1患者的基因疗法。我将在肿瘤细胞中测试这种MEN1替代病毒的有效性和安全性，然后用它来治疗小鼠模型中的肿瘤。如果研究成功，将为MEN1患者提供一种重要的新疗法。

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.