Robertson M W, Barnes M N, Rancourt C, Wang M, Grim J, Alvarez R D, Siegal G P, Curiel D T
Department of Obstetrics and Gynecology, University of Alabama at Birmingham, 35294, USA.
Semin Oncol. 1998 Jun;25(3):397-406.
Originally conceived and applied for the treatment of inherited monogenetic defects such as adenosine deaminase deficiency and cystic fibrosis, gene therapy was later applied to the treatment of cancer. Such a genetic strategy seemed rational given the recognition that cancer typically develops in a multistep process involving alterations of a number of different genes as demonstrated in familial polyposis and colorectal cancer through the work of Vogelstein et al. Because of the numerous alterations that may result in the eventual development of cancer, there is no obvious single choice for a therapeutic gene. Although one may view this as an obstacle, it also allows for a variety of possible therapeutic interventions. This review focuses on the known genetic defects that occur in ovarian cancer, the gene therapy strategies suggested by such defects, and the approaches under current development for the treatment of this disease. As such, this work also describes some of the approved human gene therapy protocols. Finally, an overview of the problems and directions for future growth and research is presented.
基因治疗最初被构思并应用于治疗诸如腺苷脱氨酶缺乏症和囊性纤维化等遗传性单基因缺陷，后来被应用于癌症治疗。鉴于人们认识到癌症通常是在一个多步骤过程中发展而来的，这个过程涉及许多不同基因的改变，就像沃格尔斯坦等人在家族性息肉病和结直肠癌研究中所证明的那样，这样一种基因策略似乎是合理的。由于可能导致癌症最终发展的改变众多，所以对于治疗基因没有明显的单一选择。尽管有人可能将此视为一个障碍，但它也允许进行各种可能的治疗干预。本综述聚焦于卵巢癌中已知的基因缺陷、由这些缺陷所提示的基因治疗策略以及当前正在开发的治疗该疾病的方法。因此，这项工作还描述了一些已获批的人类基因治疗方案。最后，对存在的问题以及未来发展和研究的方向进行了概述。
