Gene therapy for skeletal disease in MPS.

MPS 骨骼疾病的基因治疗。

• 批准号: nhmrc : 565081
• 负责人: A/Pr Donald Anson
• 金额: $ 25.59万
• 依托单位: The University of Adelaide
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2009
• 资助国家: 澳大利亚
• 起止时间: 2009-01-01 至 2011-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/gene-therapy-skeletal-disease-mps/76569
• 关键词: Gene; therapy; skeletal; disease; MPS.

Skeletal disease is a major problem for children with mucopolysaccharidoses (MPS). Patients suffer from early onset osteoporosis and osteoarthritis, severely affecting their quality of life. We will evaluate a lentiviral gene therapy vector developed in-house for its capacity to transduce bone, cartilage, synovial and ligament cells in a mouse model of MPS VI. Our goal is to generate high level, sustained expression of the deficient MPS enzyme and alter the course of skeletal disease in MPS.

皮肤病是儿童粘多糖增多症（MPS）的主要问题。患者患有早发性骨质疏松症和骨关节炎，严重影响他们的生活质量。我们将评估内部开发的慢病毒基因治疗载体在MPS VI小鼠模型中治疗骨、软骨、滑膜和韧带细胞的能力。我们的目标是产生高水平的，持续表达的缺陷MPS酶和改变过程中的MPS骨骼疾病。