Sullenger B A
Department of Experimental Surgery, Duke University Medical Center, Durham, NC, USA.
Cytokines Mol Ther. 1996 Sep;2(3):201-5.
The observation that a number of genetic mutations can result in neoplastic transformation has led many investigators to speculate that gene therapy may represent a useful approach to treat cancer. Conceptually, this application of gene therapy seems quite simple: to treat cancer, restore the correctly regulated expression of the needed tumor suppressor genes inside tumor cells and revert the transformed phenotype of such cells. However, such regulated expression has been difficult to achieve in practice. Here we describe recent efforts at such restoration via a novel approach to gene therapy that involves ribozyme-mediated repair of mutant RNA transcripts.
许多基因突变可导致肿瘤转化这一观察结果，使得许多研究人员推测基因治疗可能是一种治疗癌症的有效方法。从概念上讲，这种基因治疗的应用似乎相当简单：治疗癌症，恢复肿瘤细胞内所需肿瘤抑制基因的正确调控表达，并逆转此类细胞的转化表型。然而，这种调控表达在实践中一直难以实现。在此，我们描述了通过一种新型基因治疗方法进行这种恢复的最新努力，该方法涉及核酶介导的突变RNA转录本修复。
