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Ethical challenges for a new generation of early-phase pediatric gene therapy trials
Genetics in Medicine ( IF 8.8 ) Pub Date : 2021-07-07 , DOI: 10.1038/s41436-021-01245-3
Alexander A Iyer 1 , Dimah Saade 2 , Diana Bharucha-Goebel 2, 3 , A Reghan Foley 2 , Gilberto 'Mike' Averion 2 , Eduardo Paredes 2 , Steven Gray 4 , Carsten G Bönnemann 2 , Christine Grady 1 , Saskia Hendriks 1 , Annette Rid 1
Affiliation  

After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.



中文翻译:

新一代早期儿科基因治疗试验的伦理挑战

经过几十年的挫折,基因治疗(GT)正在经历重大突破。自 2017 年以来,五款 GT 已获得美国监管部门的批准,目前还有 900 多款正在开发中。由于缺乏有效的治疗方法,这些 GT 中的许多都针对严重限制生命的罕见儿科疾病。随着这些 GT 进入早期临床试验,具体的伦理挑战在三个领域仍未得到解决:评估风险和潜在利益、公平选择参与者以及与患者社区互动。利用我们作为临床研究人员、基础科学家和生物伦理学家参与针对一种超罕见儿科疾病的首次人体 GT 试验的经验,我们分析了这些伦理挑战,并为未来的 GT 试验提供了考虑的要点。

更新日期:2021-07-08
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