Characterisation and therapeutic intervention of two novel mouse models of Paget's disease of bone

佩吉特骨病两种新型小鼠模型的表征和治疗干预

• 批准号: G0800933/1
• 负责人: Omar Albagha
• 金额: $ 46.35万
• 依托单位: University of Edinburgh
• 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=G0800933%2F1
• 关键词: Characterisation; therapeutic; intervention; two; novel

Paget?s disease (PDB) is a common bone disease that affects about 3% of people over the age of 55 in the UK which causes pain, softening of the bones, deafness and arthritis. Genetic factors are important in the disease and two abnormal genes have been discovered. It is unclear how these faulty genes cause PDB and it is also unclear if treatment might be able to protect against disease complications if given early enough. The aim of this project is to develop an experimental model for PDB in order to find out more about why the disease occurs and to determine if early treatment might be effective in preventing complications. If our experiments are successful the information gained from these studies will be used to help design clinical trials of preventative therapy in patients with the faulty genes.

佩吉特氏病 (PDB) 是一种常见的骨病，影响英国 55 岁以上约 3% 的人，会导致疼痛、骨骼软化、耳聋和关节炎。遗传因素在该疾病中很重要，已发现两个异常基因。目前尚不清楚这些缺陷基因如何导致 PDB，也不清楚如果尽早给予治疗是否能够预防疾病并发症。该项目的目的是开发 PDB 的实验模型，以便更多地了解该疾病发生的原因，并确定早期治疗是否可以有效预防并发症。如果我们的实验成功，从这些研究中获得的信息将用于帮助设计针对有缺陷基因的患者进行预防性治疗的临床试验。