基因治疗的现状与展望

The current situation of gene therapy and its prospects

自从明确了单基因缺陷的疾病；就提出了基因治疗的概念。基因治疗是直接通过基因传递治疗人类疾病的一种手段，还可以理解成临床症状通过基因物质的传递而得到改善。目前在这个领域已经投入了几十亿美元，开展了超过三百例的临床试验，人们正通过不懈努力，期待着基因治疗的美好前景。

As soon as the first single-gene defects were define, the idea of gene therapy was proposed. Gene therapy is a therapeutic method through direct gene transfer for the treatment of human disearse. This idea has become generalized to the amelioration of any clinical symptom by the delivery of genetic material into appropriate target. More than 300 clinical trials have been carried out and with billions of dollars at stake, the field is struggling to meet expectations.