Hematology Branch, National Heart, Lung and Blood Institute, Bethesda, MD, USA.
Spark Therapeutics, Philadelphia, PA, USA.
Science. 2018 Jan 12;359(6372). doi: 10.1126/science.aan4672.
After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells. We also discuss emerging genome editing technologies that should further advance the scope and efficacy of gene therapy approaches.
经过近 30 年的曲折发展，基因疗法正迅速成为多种遗传性和获得性人类疾病治疗手段的重要组成部分。在美国和欧洲，用于遗传性免疫疾病、血友病、眼部和神经退行性疾病以及淋巴癌的基因疗法已进展到获得批准的药物地位，或有望在不久的将来获得批准。在这篇综述中，我们讨论了基因疗法发展过程中的里程碑事件，重点介绍了病毒载体的直接体内给药和经基因工程改造的 T 细胞或造血干细胞的过继转移。我们还讨论了新兴的基因组编辑技术，这些技术应该会进一步提高基因治疗方法的范围和疗效。
