Department of Pediatrics and Genetics, Stanford University, 269 Campus Drive, Room 2105, Stanford, California 94305, USA.
Nat Rev Genet. 2011 May;12(5):316-28. doi: 10.1038/nrg2971. Epub 2011 Apr 6.
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types. Several barriers need to be overcome before this type of therapy becomes a widely accepted treatment for a broad group of medical diseases. However, recent progress in the field is finally realizing some of the promises made more than 20 years ago, providing optimism for additional successes in the near future.
治疗性试验中使用的基因转移载体的改进，使得严重遗传疾病（如免疫缺陷综合征、失明和某些癌症类型）患者取得了实质性的临床成功。在这种治疗方法成为广泛接受的多种医学疾病的治疗方法之前，还需要克服一些障碍。然而，该领域最近的进展终于实现了 20 多年前做出的一些承诺，为未来取得更多成功带来了乐观情绪。
