Auman J Todd
Curr Opin Mol Ther. 2010 Dec;12(6):637-8.
Gene therapy has the potential to cure monogenic diseases through the replacement of the deleterious gene with a functional copy. While the field of gene therapy has been plagued by serious adverse events associated with therapy, it is hoped that new, safer viral vectors have reduced these risks greatly. However, recently published reports indicate that these new viral vectors are a potential risk to patients receiving gene therapy. Thus, caution is required when recruiting patients for clinical trials of gene therapies to ensure the benefit of the therapy outweighs the risks.
基因治疗有潜力通过用功能拷贝替换有害基因来治愈单基因疾病。虽然基因治疗领域一直受到与治疗相关的严重不良事件的困扰，但人们希望新的、更安全的病毒载体能大大降低这些风险。然而，最近发表的报告表明，这些新的病毒载体对接受基因治疗的患者构成潜在风险。因此，在招募患者进行基因治疗临床试验时需要谨慎，以确保治疗的益处大于风险。
