Division of Medical Ethics, NYU Grossman School of Medicine, New York, NY, USA; email:
Annu Rev Genomics Hum Genet. 2024 Aug;25(1):421-438. doi: 10.1146/annurev-genom-021623-104458.
Manipulation of a patient's genome for therapeutic ends is being attempted through numerous methods, some of which have resulted in disease-modifying interventions. The much anticipated promise of somatic gene therapy is starting to pay off; however, there remain many scientific unknowns, including concerns about safety and durability. A significant ethical concern is that of access to these novel interventions, an issue that is normally framed in terms of the high costs of approved products. I describe how access issues permeate gene therapy long before there is any commercial product and how even upstream decisions-such as choices of indication to pursue, viral vector, and where to site a trial-have significant implications for access to resultant products in both the developmental and commercial stages.
通过多种方法尝试对患者的基因组进行治疗性操作，其中一些方法已经产生了疾病修饰干预措施。体细胞基因治疗的预期承诺开始得到回报；然而，仍然存在许多科学上的未知数，包括对安全性和耐久性的担忧。一个重要的伦理问题是这些新型干预措施的可及性，这个问题通常是基于已批准产品的高昂成本来构建的。我描述了在任何商业产品出现之前，可及性问题是如何渗透到基因治疗中的，以及即使是上游决策，如选择要追求的适应症、病毒载体以及临床试验的地点，对研发和商业阶段的最终产品的可及性也有重大影响。
