siRNA therapy in dominant skin and eye disorders

siRNA 治疗主要皮肤和眼部疾病

• 批准号: G0802780/1
• 负责人: William McLean
• 金额: $ 181.79万
• 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: 2010
• 资助国家: 英国
• 起止时间: 2010 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0802780%2F1
• 关键词: siRNA; therapy; dominant; skin; eye

Incurable genetic diseases represent a major cost to the NHS, requiring specialist care costing billions per annum. RNA interference is a new Nobel prize winning technology that is suitable for treatment of a large number of genetic disorders but a major hurdle to applying this clinically is the the problem of delivery of these therapy molecules, that are slightly larger than conventional drugs, into the target tissues and organs. This programme grant focuses on a group of painful, debilitating genetic skin diseases and a related corneal eye disease, where the target tissues are very small and very accessible. The programme brings together leading researchers who uncovered the genes causing these disorders, with experts in developing new methods to deliver molecules into skin or cornea. State-of-the-art model systems will be developed to validate delivery of these new formulations into the skin and the cornea, setting the scene for taking this new therapy method into the clinic.

无法治愈的遗传疾病是NHS的主要成本，每年需要花费数十亿美元的专家护理。RNA干扰是一种新的诺贝尔奖获得技术，适用于治疗大量遗传疾病，但临床应用的主要障碍是将这些比常规药物略大的治疗分子递送到靶组织和器官中的问题。该方案赠款的重点是一组痛苦、使人衰弱的遗传性皮肤病和一种相关的角膜眼病，这些疾病的目标组织非常小，非常容易获得。该计划汇集了发现导致这些疾病的基因的领先研究人员，以及开发将分子输送到皮肤或角膜的新方法的专家。将开发最先进的模型系统，以验证这些新制剂进入皮肤和角膜的递送，为将这种新的治疗方法带入临床奠定基础。