Wolfe J H
School of Veterinary Medicine, University of Pennsylvania, Philadelphia 19104.
Curr Opin Pediatr. 1994 Apr;6(2):213-8. doi: 10.1097/00008480-199404000-00016.
The development of recombinant DNA technology, and advances in understanding molecular biology, have made it possible to alter specific functions in cells by transferring foreign genes into them. Somatic cell gene therapy for inherited diseases attempts to insert a normal copy of the mutant gene into the diseased cells of a patient to permanently correct the defect. Gene therapy has entered clinical usage, and academic medical centers are beginning to develop the specialized interdisciplinary organizations and facilities that will be needed to bring gene therapy to the bedside. Yet, the human clinical trials now underway are highly experimental because current scientific understanding of the technology is in many ways inadequate. This review highlights some of the recent progress in gene therapy for inherited disorders in animal models and human clinical trials.
重组DNA技术的发展以及对分子生物学认识的进步，使得通过将外源基因导入细胞来改变细胞的特定功能成为可能。针对遗传性疾病的体细胞基因治疗试图将突变基因的正常拷贝插入患者的患病细胞中，以永久性地纠正缺陷。基因治疗已进入临床应用阶段，学术医疗中心也开始建立将基因治疗应用于临床所需的专门跨学科组织和设施。然而，目前正在进行的人体临床试验具有高度的实验性，因为目前对该技术的科学理解在很多方面还不够充分。本综述重点介绍了在动物模型和人体临床试验中遗传性疾病基因治疗方面的一些最新进展。
