OPTIMISED DELIVERY OF THERAPEUTIC siRNA INTO HUMAN SKIN

RNAi是一种新的治疗形式，可以在广泛的遗传病和癌症中关闭有缺陷的基因。应用这种革命性的新药物的障碍是如何将其输送到人体内，因为涉及的分子比正常药物更大，因此细胞需要一些帮助来吸收这种材料，才能产生有益的效果。该项目的主要目的是革命性地将这些新疗法作为一种测试器官系统送入皮肤。皮肤的优势是在身体外面，很容易接触到，而且整容手术(乳房缩小和腹部整形)产生的多余皮肤很容易用于实验，以优化RNAi分子的传递。为了实现这一目标，我们将开发一种创新的新工具，将化学、超声波和显微无痛针阵列结合在一起，以快速、有效和可靠的方式将药物输送到皮肤中，覆盖率高。一旦针对皮肤病进行了优化，这种手持设备将很容易通过锁孔手术方法在内部器官上使用，因此这种设备在所有医学分支中的潜在应用都是巨大的。在邓迪集合的团队将领先的分子生物学家和工程师聚集在一起，在许多不同领域拥有不同技能的更多国际合作者的支持下，以前所未有的方式拼凑技术元素。据估计，这里正在解决的治疗提供问题的市场规模约为每年数百亿美元。

RNAi is a new form of therapy that allows faulty genes to be switched off in a wide range of genetic diseases and in cancer. The barrier to applying this revolutionary new form of medicine is how to deliver it into the body, since the molecules involved are larger than normal drugs and so cells require some help in taking this material up so it can have its beneficial effect. The main aim of this project is to revolutionise the delivery of these new therapeutics into the skin, as a test organ system. Skin has the advantage of being on the outside of the body for easy access, and excess human skin from cosmetic surgery (breast reductions and tummy tucks) is readily available for experimentation to optimise delivery of RNAi molecules. In pursuit of this, we will develop an innovative new tool combining chemistry, ultrasound and arrays of microscopic painless needles for fast, effective and reliable delivery into skin at high coverage rates. Once optimised for skin diseases, this hand-held device will be readily adapted for use on internal organs using keyhole surgery methods, so that the potential applications of this device across all branches of medicine are huge. The team assembled in Dundee brings together leading molecular biologists with engineers, supported by further international collaborators with diverse skills across many different fields, in order to piece together elements of technology in ways that have never been done before. The estimated market for solving the therapy delivery problem being tackled here is in the order of 10s of billions of dollars per year.