肝脏靶向基因治疗。

Liver-directed gene therapy.

作者信息

Nunes F A, Raper S E

机构信息

Division of Gastroenterology, University of Pennslyvania School of Medicine, Philadelphia 19104, USA.

出版信息

Med Clin North Am. 1996 Sep;80(5):1201-13. doi: 10.1016/s0025-7125(05)70486-7.

DOI:10.1016/s0025-7125(05)70486-7

PMID:8804381

Abstract

Liver-directed gene therapy represents a promising new modality for the treatment of inherited and acquired liver diseases. Clinical trials of liver-directed gene therapy are underway for diseases such as FH, OTC deficiency, and cancer. The main obstacles to effective gene therapy are the limitations of present gene delivery technology to express a desired gene safely and stably at therapeutic levels. With improved gene delivery technology and refinements in the ex vivo and in vivo approaches, a truly useful clinical tool will emerge.

摘要

肝脏定向基因治疗是一种治疗遗传性和获得性肝脏疾病的有前景的新方法。针对家族性高胆固醇血症（FH）、鸟氨酸氨甲酰基转移酶缺乏症（OTC缺乏症）和癌症等疾病的肝脏定向基因治疗临床试验正在进行。有效基因治疗的主要障碍是当前基因递送技术在治疗水平上安全稳定地表达所需基因的局限性。随着基因递送技术的改进以及体外和体内方法的完善，一种真正有用的临床工具将会出现。