Biological membrane transporters: Delivery of an oligonucleotide inhibitor of vascular endothelial growth factor (VEGF)

脉络膜新生血管是年龄相关性黄斑变性最严重的形式，是发达国家失明的主要原因。如果能够解决与DNA传递相关的问题，基因疗法可能是治疗这种疾病的一种方法。我们的项目涉及一种高度新颖的基因传递策略，涉及亲脂树状大分子（表面带正电荷的树状化合物）离子对的形成。我们将开发新的dna -树状大分子复合物，并在一个完善的动物模型中测试它们的新生血管。该项目的成功完成可能为脉络膜新生血管提供一种潜在的治疗方法，并有很好的机会进入人体临床试验。

Choroidal neovascularisation, which is the most severe form of Age Related Macular Degeneration, is the major cause of blindness in the developed world. Gene therapy could be a cure for this disease if the problems associated with the delivery of DNA could be addressed. Our project involves a highly novel strategy for gene delivery involving ion pair formation of lipophilic dendrimers (tree-like compounds with positive charges on the surface). We will develop new DNA-dendrimer complexes and test them in a well established animal model for neovascularisation. Successful completion of this project might offer a potential therapy for choroidal neovascularisation, with a good chance of entering into human clinical trials.