San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, 20132 Milan, Italy.
Vita Salute San Raffaele University, 20132 Milan, Italy.
Nature. 2015 Oct 15;526(7573):351-60. doi: 10.1038/nature15818.
Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver 'cures' for some otherwise terminal or severely disabling conditions. Behind these advances lie improved vector designs that enable the safe delivery of therapeutic genes to specific cells. Technologies for editing genes and correcting inherited mutations, the engagement of stem cells to regenerate tissues and the effective exploitation of powerful immune responses to fight cancer are also contributing to the revitalization of gene therapy.
最近的基因治疗临床试验显示出显著的治疗效果和极好的安全性记录。它们为基因治疗长期以来的承诺提供了证据，即可以为某些原本致命或严重致残的疾病提供“治愈”方法。这些进展背后是改进的载体设计，能够安全地将治疗基因递送到特定的细胞。编辑基因和纠正遗传突变的技术、利用干细胞来再生组织以及有效地利用强大的免疫反应来对抗癌症的技术也为基因治疗的复兴做出了贡献。
