Potential implications of cell therapy for osteogenesis imperfecta.
Potential implications of cell therapy for osteogenesis imperfecta.
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DOI：
10.2217/17584272.4.1.57
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发表时间：
2009-02-01
期刊：
影响因子：
--
通讯作者：
Li F
中科院分区：
文献类型：
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作者：
Niyibizi C;Li F
成骨不全（OI）是一种以骨脆性为标志的脆性骨疾病。由于这种疾病是遗传性的，目前还没有可用的治疗方法。除了最近使用的双膦酸盐外，已经尝试了几种药物，但没有取得多大成功。干细胞已被建议作为一种替代性的OI治疗方法，但在这项技术应用于治疗OI患者之前，仍存在许多障碍。本文综述了目前已知的干细胞应用于治疗OI的动物模型，使用间充质干细胞治疗OI患者的临床试验和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。要克服的障碍，使干细胞接近临床和未来的前景进行了讨论。
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.