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首页> 外文期刊>Frontiers in neuroendocrinology >Gene therapy for pituitary tumors: from preclinical models to clinical implementation.
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Gene therapy for pituitary tumors: from preclinical models to clinical implementation.

机译:垂体肿瘤的基因治疗:从临床前模型到临床实施。

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摘要

Gene therapy, which entails the use of nucleic acids as drugs, is a new approach to treat disease. Gene therapy has been successfully implemented in several preclinical animal models, including several paradigms of experimental pituitary tumors. In spite of these successes, several critical issues need to be addressed before gene therapy can become a clinical reality for the treatment of pituitary tumors. These include the development of safer and more effective gene delivery vectors, the uncovering of novel therapeutic targets, the development of molecular switches which will allow turning therapeutic transgene expression "on" and "off" as and when it is needed, and the ability to scale up the vector preparations devoid of any putative contaminants. There are still many basic science developments that must take place in order to allow this new therapeutic technology to make its way successfully into the clinical arena to treat pituitary disease. We envisage these developments taking place within the next five years, gene therapy for pituitary tumors will then form part of the armamentarium available to better treat and manage pituitary tumors.
机译:基因疗法需要使用核酸作为药物,是治疗疾病的新方法。基因治疗已经在几种临床前动物模型中成功实施,包括实验性垂体瘤的几种范例。尽管取得了这些成功,在基因治疗成为治疗垂体肿瘤的临床现实之前,仍需要解决几个关键问题。其中包括开发更安全,更有效的基因递送载体,发现新的治疗靶标,开发分子开关,这些开关将使治疗性转基因表达在需要时“开启”和“关闭”,以及扩大不含任何假定污染物的载体制剂。为了使这种新的治疗技术成功进入垂体疾病的临床治疗领域,仍然必须进行许多基础科学开发。我们预计这些发展将在未来五年内发生,垂体肿瘤的基因治疗将成为可用于更好地治疗和管理垂体肿瘤的武器库的一部分。

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