Niyibizi Christopher, Li Feng
Int J Clin Rheumtol. 2009 Feb 1;4(1):57-66. doi: 10.2217/17584272.4.1.57.
Osteogenesis imperfecta (OI) is a brittle-bone disease whose hallmark is bone fragility. Since the disease is genetic, there is currently no available cure. Several pharmacological agents have been tried with not much success, except the recent use of bisphosphonates. Stem cells have been suggested as an alternative OI treatment, but many hurdles remain before this technology can be applied for treating patients with OI. This review summarizes what is known at present regarding the application of stem cells to treat OI using animal models, clinical trials using mesenchymal stem cells to treat patients with OI and the knowledge gained from the clinical trials. Application of gene therapy in combination with stem cells is also discussed. The hurdles to be overcome to bring stem cells close to the clinic and future perspectives are discussed.
成骨不全症（OI）是一种以骨骼脆弱为特征的脆骨病。由于该疾病是遗传性的，目前尚无可用的治愈方法。除了最近使用双膦酸盐外，已经尝试了几种药物，但效果不太理想。干细胞已被提议作为一种治疗OI的替代方法，但在该技术可应用于治疗OI患者之前，仍有许多障碍。本综述总结了目前关于使用动物模型将干细胞应用于治疗OI、使用间充质干细胞治疗OI患者的临床试验以及从临床试验中获得的知识。还讨论了基因治疗与干细胞联合应用的情况。讨论了使干细胞接近临床所需克服的障碍以及未来前景。
