Piccolo Pasquale, Brunetti-Pierri Nicola
1 Telethon Institute of Genetics and Medicine , Pozzuoli, Naples 80078, Italy .
Hum Gene Ther. 2015 Apr;26(4):186-92. doi: 10.1089/hum.2015.029. Epub 2015 Apr 1.
Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.
基因治疗正进入初始临床开发阶段，以治疗越来越多的遗传性代谢疾病。本综述概述了针对主要在肝脏中表达的酶缺乏所致疾病的肝脏定向基因治疗的发展，并讨论了在临床转化方面最具前景的疾病。
