多关节炎性疾病、损伤或退变的共同特点是关节软骨的破坏或磨损，是导致肢体废用的主要原因，其特点在于受累关节的多分布性。单部位治疗手段其效果较为有限，而系统性基因治疗是富有挑战性和广阔前景的前沿干预方式，尤其适用于多发关节软骨病损。本课题以灵长类动物为研究对象，选取兼有抗炎抑制破坏以及促软骨细胞增殖和基质合成作用的目的基因协同起效，利用安全性好、转染效率更高的LPD非病毒转染系统载体，以分化能力强、免疫耐受好的hUCB－MSCs介导，并从载体表面特异性抗体修饰获得免疫寻靶功能、原始干细胞归巢性能以及局部超声空化效应等多方面增强系统性基因治疗的靶向性和表达效率，将目的基因引导到达多处病变软骨部位进行表达，产生单次应用多点表达的效果。将上述诸因素进行有效整合，可在多部位关节软骨产生积极的生物学响应，有望对多部位关节病损皆出现有效的治疗效果。

The common characteristics of the multi-joint disease, injury or degeneration are the erosion or attrition of hyaline cartilage, which is the main reason for disability of extremities. Treatment on single joint is limit for such pathological change relate to multiple joints. Systemic gene therapy is the cutting-edge intervention rich in challenge and broad prospects, especially for the cartilage in multiple joints. In this research, primates were selected for detection of a combined systemic gene therapy to treat cartilage erosion in involved joints. Target genes with the features of anti-inflammatory and restrain degeneration, or promote the proliferation and matrix synthesis of chondrocytes act together within the hUCB-MSCs, which possess the advantages of powerful differentiate ability and prominent immune tolerance. The safer LPD non-viral transfection vector, with higher transfect efficiency, was used to enhance the level of targeted cytokine expression. Immune targeting function will be acquired by the modification of specific antibody on the surface of the vector. Targeting and express efficiency of the systemic gene therapy will also be augmented by homing property of primordial stem cells and local ultrasonic cavitation effect. The target genes will be guided to arrive at multiple sites with cartilage erosion and express in situ. By this means, single application will generate multiple expressions. By integration of these factors, positive biological response of cartilage will be aroused within multiple joints, so as to arise the effective treatment.