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用于治疗垂体肿瘤的基因疗法。

GENE THERAPY FOR THE TREATMENT OF PITUITARY TUMORS.

作者信息

Rodriguez Silvia S, Castro Maria G, Brown Oscar A, Goya Rodolfo G, Console Gloria M

机构信息

Histology and Embryology B-CICPBA.

出版信息

Expert Rev Endocrinol Metab. 2009 Jul 1;4(4):359-370. doi: 10.1586/eem.09.16.

Abstract

Pituitary adenomas constitute the most frequent neuroendocrine pathology in humans. Current therapies include surgery, radiotherapy and pharmacological approaches. Although useful, none of them offers a permanent cure. Current research efforts to implement gene therapy in pituitary tumors include the treatment of experimental adenomas with adenoviral vector-mediated transfer of the suicide gene for thymidine kinase, which converts the prodrug ganciclovir into a toxic metabolite. In some cases, the suicide transgene has been placed under the control of pituitary cell-type specific promoters. Also, regulatable adenoviral vector systems are being assessed in gene therapy approaches for experimental pituitary tumors. Although the efficiency and safety of current viral vectors must be optimized before clinical use, they remain as highly promising therapeutic tools.

摘要

垂体腺瘤是人类最常见的神经内分泌疾病。目前的治疗方法包括手术、放疗和药物治疗。尽管这些方法都有用,但没有一种能提供永久性治愈。目前在垂体肿瘤中实施基因治疗的研究工作包括用腺病毒载体介导的胸苷激酶自杀基因转移来治疗实验性腺瘤,该基因可将前体药物更昔洛韦转化为有毒代谢物。在某些情况下,自杀转基因已置于垂体细胞类型特异性启动子的控制之下。此外,可调节腺病毒载体系统正在用于实验性垂体肿瘤的基因治疗方法中进行评估。尽管目前的病毒载体在临床使用前必须优化其效率和安全性,但它们仍然是非常有前景的治疗工具。

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