基因治疗产品的开发现状与挑战

基因治疗是将外源性遗传物质通过多种方法转移到靶细胞中,用来治疗特定疾病。基因治疗对某些单基因遗传病有一次治愈的可能,或将成为未来治疗人类疾病的重要手段。但基因治疗仍存在诸多挑战,如长期的安全性和疗效数据、可及性以及监管等。从基因治疗的基本概念和历史出发,结合基因治疗产品的开发现状和未来,进行讨论。

Current Status and Challenges of Gene Therapy Products

Gene therapy is an approach treating certain diseases by transferring therapeutic genetic material to target cells. Gene therapy may provide the possibility of “one-time” cure paving the way as an important treatment armamentarium in near future. However, challenges subsuming long-term efficacy and safety data, accessibility, and regulatory policy forgene therapy need to be addressed. The review will start with basic concept and history, to discuss current status and future prospects of gene therapy.