Hassan Memy H, Othman Essam E, Hornung Daniela, Al-Hendy Ayman
Department of Pharmacology and Toxicology, Faculty of Pharmacy, Al-Azhar University, Cairo, Egypt.
Adv Drug Deliv Rev. 2009 Aug 10;61(10):822-35. doi: 10.1016/j.addr.2009.04.023. Epub 2009 May 13.
Gene therapy is the introduction of genetic material into patient's cells to achieve therapeutic benefit. Advances in molecular biology techniques and better understanding of disease pathogenesis have validated the use of a variety of genes as potential molecular targets for gene therapy based approaches. Gene therapy strategies include: mutation compensation of dysregulated genes; replacement of defective tumor-suppressor genes; inactivation of oncogenes; introduction of suicide genes; immunogenic therapy and antiangiogenesis based approaches. Preclinical studies of gene therapy for various gynecological disorders have not only shown to be feasible, but also showed promising results in diseases such as uterine leiomyomas and endometriosis. In recent years, significant improvement in gene transfer technology has led to the development of targetable vectors, which have fewer side-effects without compromising their efficacy. This review provides an update on developing gene therapy approaches to treat common gynecological diseases such as uterine leiomyoma and endometriosis.
基因治疗是将遗传物质导入患者细胞以实现治疗益处。分子生物学技术的进步以及对疾病发病机制的更好理解已证实，多种基因可作为基于基因治疗方法的潜在分子靶点。基因治疗策略包括：对失调基因进行突变补偿；替换有缺陷的肿瘤抑制基因；使癌基因失活；引入自杀基因；免疫治疗和基于抗血管生成的方法。针对各种妇科疾病的基因治疗临床前研究不仅已证明是可行的，而且在子宫肌瘤和子宫内膜异位症等疾病中也显示出了有前景的结果。近年来，基因转移技术的显著改进导致了可靶向载体的开发，这些载体副作用较少且不影响其疗效。本综述提供了关于治疗子宫肌瘤和子宫内膜异位症等常见妇科疾病的基因治疗方法进展的最新情况。
