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首页> 外文期刊>Journal of Cellular Physiology >Orthopedic gene therapy--lost in translation?
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Orthopedic gene therapy--lost in translation?

机译:基因治疗骨科,迷失在翻译?

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摘要

Orthopedic gene therapy has been the topic of considerable research for two decades. The preclinical data are impressive and many orthopedic conditions are well suited to genetic therapies. But there have been few clinical trials and no FDA-approved product exists. This paper examines why this is so. The reasons are multifactorial. Clinical translation is expensive and difficult to fund by traditional academic routes. Because gene therapy is viewed as unsafe and risky, it does not attract major funding from the pharmaceutical industry. Start-up companies are burdened by the complex intellectual property environment and difficulties in dealing with the technology transfer offices of major universities. Successful translation requires close interactions between scientists, clinicians and experts in regulatory and compliance issues. It is difficult to create such a favorable translational environment. Other promising fields of biological therapy have contemplated similar frustrations approximately 20 years after their founding, so there seem to be more general constraints on translation that are difficult to define. Gene therapy has noted some major clinical successes in recent years, and a sense of optimism is returning to the field. We hope that orthopedic applications will benefit collaterally from this upswing and move expeditiously into advanced clinical trials.
机译:基因治疗骨科的话题相当大的研究二十年。临床前数据令人印象深刻,许多骨科条件适合的基因疗法。试验和fda批准的产品不存在。论文调查了为什么会这样。多因素疾病。和难以通过传统学术基金路线。和风险,它不吸引大资金制药行业。由复杂的知识产权负担在处理环境和困难技术转移的主要办公室大学。科学家之间的相互作用,临床医生和专家在监管和合规问题。很难创建这样一个有利的转化环境。生物治疗的考虑相似挫折后大约20年成立,所以似乎更普遍限制翻译很难定义的。近年来临床成功和有意义乐观是返回的字段。骨科应用程序将会受益种间接从本轮复苏中移动迅速地进入先进的临床试验。

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