Somatic Gene Therapy: Ethics and Access.

IF 7.7 2区 生物学 Q1 GENETICS & HEREDITY
Alison Bateman-House
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引用次数: 0

Abstract

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.

体细胞基因疗法:伦理与获取。
目前,人们正尝试通过多种方法操纵患者的基因组以达到治疗目的,其中一些方法已经产生了改变疾病的干预效果。体细胞基因疗法的前景令人期待,目前已初见成效;然而,科学上仍有许多未知数,包括对安全性和持久性的担忧。一个重要的伦理问题是如何获得这些新型干预措施,而这个问题通常被归结为获批产品的高昂成本。我将描述在基因疗法还没有任何商业产品之前,获取问题是如何渗透其中的,以及即使是上游决策--如选择适应症、病毒载体和试验地点--是如何对研发和商业阶段的成果产品的获取产生重大影响的。
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来源期刊
CiteScore
14.90
自引率
1.10%
发文量
29
期刊介绍: Since its inception in 2000, the Annual Review of Genomics and Human Genetics has been dedicated to showcasing significant developments in genomics as they pertain to human genetics and the human genome. The journal emphasizes genomic technology, genome structure and function, genetic modification, human variation and population genetics, human evolution, and various aspects of human genetic diseases, including individualized medicine.
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