Kone B C
Department of Internal Medicine, The University of Texas Medical School at Houston, 77030, USA.
Semin Nephrol. 2000 Jan;20(1):47-59.
Nephrology is entering the age of genomics-based drug discovery and development. Once only a theoretical objective, gene therapy is now being tested in various diseases. New and substantially improved vector systems and related technologies are undergoing development, many have shown promise in animal studies, and some are now being used in clinical trials. Recent advances in the molecular basis for renal diseases, organ transplant rejection, and hypertension have led to preclinical tests of gene therapeutic approaches. The most impressive of these strategies will likely soon be studied in the clinic. This review details recent advances in gene therapy technology and highlights potential novel applications of gene therapy in the treatment of renal diseases and hypertension. While the manufacture and widespread use of gene therapy products as conventional pharmaceuticals for renal diseases and hypertension may seem to be a goal for the remote future, much of the needed genetic information, technology, and intellectual resources are rapidly becoming available.
肾脏病学正在步入基于基因组学的药物发现与开发时代。基因治疗曾经只是一个理论目标，如今正在多种疾病中进行试验。新型且有显著改进的载体系统及相关技术正在研发中，许多已在动物研究中展现出前景，有些目前正在进行临床试验。肾脏疾病、器官移植排斥反应和高血压分子基础方面的最新进展已促使对基因治疗方法进行临床前测试。这些策略中最令人瞩目的可能很快就会进入临床研究。本综述详细介绍了基因治疗技术的最新进展，并突出了基因治疗在肾脏疾病和高血压治疗中的潜在新应用。虽然将基因治疗产品作为治疗肾脏疾病和高血压的常规药物进行生产和广泛使用似乎是遥远未来的目标，但许多所需的遗传信息、技术和智力资源正迅速可得。
