Obermiller P S, Tait D L, Holt J T
Vanderbilt University Cancer Center, 2220 Pierce Avenue South, Nashville, TN 37232, USA.
Breast Cancer Res. 2000;2(1):28-31. doi: 10.1186/bcr26. Epub 1999 Dec 17.
Gene therapy is a therapeutic approach that is designed to correct specific molecular defects that contribute to the cause or progression of cancer. Genes that are mutated or deleted in cancers include the cancer susceptibility genes p53 and BRCA1. Because mutational inactivation of gene function is specific to tumor cells in these settings, cancer gene correction strategies may provide an opportunity for selective targeting without significant toxicity for normal nontumor cells. Both p53 and BRCA1 appear to inhibit cancer cells that lack mutations in these genes, suggesting that the so-called gene correction strategies may have broader potential than initially believed. Increasing knowledge of cancer genetics has identified these and other genes as potential targets for gene replacement therapy. Initial patient trials of p53 and BRCA1 gene therapy have provided some indications of potential efficacy, but have also identified areas of basic and clinical research that are needed before these approaches may be widely used in patient care.
基因治疗是一种治疗方法，旨在纠正导致癌症发生或进展的特定分子缺陷。在癌症中发生突变或缺失的基因包括癌症易感基因p53和BRCA1。由于在这些情况下基因功能的突变失活是肿瘤细胞特有的，癌症基因校正策略可能为选择性靶向提供机会，而对正常非肿瘤细胞没有明显毒性。p53和BRCA1似乎都能抑制这些基因无突变的癌细胞，这表明所谓的基因校正策略可能比最初认为的具有更广泛的潜力。对癌症遗传学的了解不断增加，已将这些基因和其他基因确定为基因替代疗法的潜在靶点。p53和BRCA1基因治疗的初步患者试验已提供了一些潜在疗效的迹象，但也确定了在这些方法可广泛应用于患者护理之前所需的基础和临床研究领域。
