Sandhu J S, Keating A, Hozumi N
Department of Surgery, University of Toronto, Canada.
Crit Rev Biotechnol. 1997;17(4):307-26. doi: 10.3109/07388559709146617.
Human gene therapy and its application for the treatment of human genetic disorders, such as cystic fibrosis, cancer, and other diseases, are discussed. Gene therapy is a technique in which a functioning gene is inserted into a human cell to correct a genetic error or to introduce a new function to the cell. Many methods, including retroviral vectors and non-viral vectors, have been developed for both ex vivo and in vivo gene transfer into cells. Vectors need to be developed that efficiently transfer genes to target cells, and promoter systems are required that regulate gene expression according to physiologic needs of the host cell. There are several safety and ethical issues related to manipulating the human genome that need to be resolved. Current gene therapy efforts focus on gene insertion into somatic cells only. Gene therapy has potential for the effective treatment of genetic disorders, and gene transfer techniques are being used for basic research, for example, in cancer, to examine the underlying mechanism of disease. There are still many technical obstacles to be overcome before human gene therapy can become a routine procedure. The current human genome project provides the sequences of a vast number of human genes, leading to the identification, characterization, and understanding of genes that are responsible for many human diseases.
本文讨论了人类基因治疗及其在治疗人类遗传疾病（如囊性纤维化、癌症和其他疾病）中的应用。基因治疗是一种将功能正常的基因插入人体细胞以纠正基因错误或为细胞引入新功能的技术。已经开发了许多方法，包括逆转录病毒载体和非病毒载体，用于体外和体内的细胞基因转移。需要开发能够有效将基因转移到靶细胞的载体，并且需要根据宿主细胞的生理需求调节基因表达的启动子系统。与操纵人类基因组相关的几个安全和伦理问题需要解决。目前的基因治疗仅专注于将基因插入体细胞。基因治疗有有效治疗遗传疾病的潜力，并且基因转移技术正用于基础研究，例如在癌症研究中，以研究疾病的潜在机制。在人类基因治疗成为常规程序之前，仍有许多技术障碍需要克服。当前的人类基因组计划提供了大量人类基因的序列，从而导致对许多人类疾病相关基因的鉴定、表征和理解。
