OPTIMISED DELIVERY OF THERAPEUTIC siRNA INTO HUMAN SKIN

优化将治疗性 siRNA 输送到人体皮肤中

• 批准号: G0801742/1
• 负责人: William McLean
• 金额: $ 66.13万
• 依托单位: University of Dundee
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2009
• 资助国家: 英国
• 起止时间: 2009 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0801742%2F1
• 关键词: OPTIMISED; DELIVERY; THERAPEUTIC; siRNA; INTO

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.

RNAi 是一种新的疗法，可以在多种遗传疾病和癌症中关闭有缺陷的基因。应用这种革命性的新型药物的障碍是如何将其输送到体内，因为所涉及的分子比普通药物大，因此细胞需要一些帮助来吸收这种材料，以便发挥其有益作用。该项目的主要目标是彻底改变将这些新疗法作为测试器官系统传递到皮肤中的方式。皮肤的优点是位于体外，易于接触，整容手术（乳房缩小术和腹部除皱术）中多余的人体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，结合化学、超声波和显微无痛针阵列，以高覆盖率快速、有效和可靠地输送到皮肤中。一旦针对皮肤疾病进行了优化，这种手持式设备将很容易适用于使用锁孔手术方法治疗内脏器官，因此该设备在所有医学分支的潜在应用都是巨大的。在邓迪组建的团队汇集了领先的分子生物学家和工程师，并得到了跨多个不同领域的不同技能的国际合作者的支持，以便以前所未有的方式将技术要素拼凑在一起。估计用于解决这里正在解决的治疗传递问题的市场每年约为数十亿美元。