Prenatal gene editing for neurodevelopmental diseases: Ethical considerations.

IF 8.1 1区 生物学 Q1 GENETICS & HEREDITY
American journal of human genetics Pub Date : 2025-02-06 Epub Date: 2025-01-28 DOI:10.1016/j.ajhg.2025.01.003
Rami M Major, Eric T Juengst
{"title":"Prenatal gene editing for neurodevelopmental diseases: Ethical considerations.","authors":"Rami M Major, Eric T Juengst","doi":"10.1016/j.ajhg.2025.01.003","DOIUrl":null,"url":null,"abstract":"<p><p>Neurodevelopmental diseases (NDDs) are notoriously difficult to treat because clinical symptoms stem from developmental processes that begin before birth. Prenatal gene editing could fill the treatment gap for NDDs by targeting and permanently correcting the genetic variants that underlie these pathogenic developmental processes. At the same time, there is a risk of unintended edits to the fetus or the pregnant person that could result in serious adverse consequences that are difficult, if not impossible, to undo. This raises ethical concerns that make the development of prenatal gene editing especially challenging. To date, there are no frameworks for considering the steps necessary for an ethical path forward for prenatal gene editing specifically. The 60-year history of in utero therapy has included the development of frameworks for other therapies that can provide starting points for addressing the unique issues of prenatal gene editing. We identified 12 themes from 17 ethical frameworks, literature, consensus statements, and government reports on prenatal interventions that could set precedents for prenatal gene editing interventions. In considering these alongside current criteria for postnatal gene therapies for NDDs, we discuss a path forward for prenatal gene editing interventions of NDDs.</p>","PeriodicalId":7659,"journal":{"name":"American journal of human genetics","volume":" ","pages":"201-214"},"PeriodicalIF":8.1000,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11866956/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"American journal of human genetics","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1016/j.ajhg.2025.01.003","RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/28 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0

Abstract

Neurodevelopmental diseases (NDDs) are notoriously difficult to treat because clinical symptoms stem from developmental processes that begin before birth. Prenatal gene editing could fill the treatment gap for NDDs by targeting and permanently correcting the genetic variants that underlie these pathogenic developmental processes. At the same time, there is a risk of unintended edits to the fetus or the pregnant person that could result in serious adverse consequences that are difficult, if not impossible, to undo. This raises ethical concerns that make the development of prenatal gene editing especially challenging. To date, there are no frameworks for considering the steps necessary for an ethical path forward for prenatal gene editing specifically. The 60-year history of in utero therapy has included the development of frameworks for other therapies that can provide starting points for addressing the unique issues of prenatal gene editing. We identified 12 themes from 17 ethical frameworks, literature, consensus statements, and government reports on prenatal interventions that could set precedents for prenatal gene editing interventions. In considering these alongside current criteria for postnatal gene therapies for NDDs, we discuss a path forward for prenatal gene editing interventions of NDDs.

神经发育疾病的产前基因编辑:伦理考虑。
众所周知,神经发育疾病(ndd)很难治疗,因为临床症状源于出生前就开始的发育过程。产前基因编辑可以通过靶向和永久纠正这些致病发育过程背后的遗传变异来填补ndd的治疗空白。与此同时,存在对胎儿或孕妇进行意外编辑的风险,这可能导致严重的不良后果,即使不是不可能,也很难消除。这引发了伦理问题,使产前基因编辑的发展尤其具有挑战性。迄今为止,还没有专门考虑产前基因编辑伦理道路所需步骤的框架。子宫内治疗的60年历史包括其他治疗框架的发展,这些治疗框架可以为解决产前基因编辑的独特问题提供起点。我们从17个伦理框架、文献、共识声明和政府关于产前干预的报告中确定了12个主题,这些主题可以为产前基因编辑干预设定先例。在考虑这些以及ndd产后基因治疗的当前标准时,我们讨论了ndd产前基因编辑干预的前进道路。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
CiteScore
14.70
自引率
4.10%
发文量
185
审稿时长
1 months
期刊介绍: The American Journal of Human Genetics (AJHG) is a monthly journal published by Cell Press, chosen by The American Society of Human Genetics (ASHG) as its premier publication starting from January 2008. AJHG represents Cell Press's first society-owned journal, and both ASHG and Cell Press anticipate significant synergies between AJHG content and that of other Cell Press titles.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信