Gene therapy for the treatment of musculoskeletal diseases
Gene therapy for the treatment of musculoskeletal diseases
复制标题
DOI：
10.5435/00124635-200507000-00003
复制
发表时间：
2005-07-01
影响因子：
3.2
通讯作者：
Robbins, PD
中科院分区：
文献类型：
--
作者：
Evans, CH;Ghivizziani, SC;Robbins, PD
基因疗法在骨科应用的研究在治疗慢性和急性遗传性和非遗传性疾病方面取得了进展。关节炎的基因治疗是最初的研究重点，目前已启动多项一期临床试验。初步研究结果支持基因疗法在治疗其他慢性疾病中的应用，包括骨质疏松症和无菌性松动以及肌肉骨骼肿瘤。最快的进展可能是组织修复，因为它既不需要长期的转基因表达，也不需要严格调控的转基因表达水平。此外，利用现有技术或许可以实现治愈。在临床前研究中，骨愈合的基因调节刺激在修复长骨和颅骨的节段性缺陷以及提高脊柱融合的成功率方面显示出令人印象深刻的结果。越来越多的证据表明，基因转移可以帮助修复关节软骨、半月板、椎间盘、韧带和肌腱。这些发展有可能改变肌肉骨骼护理的许多领域，从而带来比现有治疗方式侵入性更小、更有效且更便宜的治疗方法。
Research into the orthopaedic applications of gene therapy has resulted in progress toward managing chronic and acute genetic and nongenetic disorders. Gene therapy for arthritis, the original focus of research, has progressed to the initiation of several phase 1 clinical trials. Preliminary findings support the application of gene therapy in the treatment of additional chronic conditions, including osteoporosis and aseptic loosening, as well as musculoskeletal tumors. The most rapid progress is likely to be in tissue repair because it requires neither long-term transgene expression nor closely regulated levels of transgene expression. Moreover, healing probably can be achieved with existing technology. In preclinical studies, genetically modulated stimulation of bone healing has shown impressive results in repairing segmental defects in the long bones and cranium and in improving the success of spinal fusions. An increasing amount of evidence indicates that gene transfer can aid the repair of articular cartilage, menisci, intervertebral disks, ligaments, and tendons. These developments have the potential to transform many areas of musculoskeletal care, leading to treatments that are less invasive, more effective, and less expensive than existing modalities.