{"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.
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.