State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.

ABSTRACT: In Part I of this Review (Wang and Gao, 2014), we introduced recent advances in gene delivery technologies and explained how they have powered some of the current human gene therapy applications. In Part II, we expand the discussion on gene therapy applications, focusing on some of the most exciting clinical uses. To help readers to grasp the essence and to better organize the diverse applications, we categorize them under four gene therapy strategies: (1) gene replacement therapy for monogenic diseases, (2) gene addition for complex disorders and infectious diseases, (3) gene expression alteration targeting RNA, and (4) gene editing to introduce targeted changes in host genome. Human gene therapy started with the simple idea that replacing a faulty gene with a functional copy can cure a d

SUBMITTER: Wang D 

PROVIDER: S-EPMC4440458 | biostudies-literature | 2014 Sep

REPOSITORIES: biostudies-literature