{"title":"In Utero Therapies, the Next Frontier.","authors":"Teodora R Kolarova, Edith Cheng","doi":"10.1002/ajmg.c.32135","DOIUrl":null,"url":null,"abstract":"<p><p>Advancements in molecular diagnostics and the expanded understanding of pathophysiologic processes underlying a variety of genetic conditions have led to the design and implementation of many targeted therapies in the past decade. In the prenatal space, these advancements have rapidly changed the field of prenatal diagnosis and have garnered enthusiasm toward interventions aimed at correcting specific disease mechanisms in utero to prevent irreversible injury and/or leverage fetal physiology to increase the effectiveness of these treatments. Although many promising trials are underway, continued efforts to fully elucidate the intricacies of fetal immunology, the fetal blood-brain barrier and precise molecular processes during different stages of development will be paramount to continued refinement of in utero therapies. The social and ethical implications of fetal therapy are also of utmost importance, especially in an era of increasing restrictions on reproductive autonomy. This review aims to summarize current efforts toward in utero targeted fetal therapies as well as the history and ethical implications of such endeavors.</p>","PeriodicalId":7445,"journal":{"name":"American Journal of Medical Genetics Part C: Seminars in Medical Genetics","volume":" ","pages":"e32135"},"PeriodicalIF":2.8000,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"American Journal of Medical Genetics Part C: Seminars in Medical Genetics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ajmg.c.32135","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0
Abstract
Advancements in molecular diagnostics and the expanded understanding of pathophysiologic processes underlying a variety of genetic conditions have led to the design and implementation of many targeted therapies in the past decade. In the prenatal space, these advancements have rapidly changed the field of prenatal diagnosis and have garnered enthusiasm toward interventions aimed at correcting specific disease mechanisms in utero to prevent irreversible injury and/or leverage fetal physiology to increase the effectiveness of these treatments. Although many promising trials are underway, continued efforts to fully elucidate the intricacies of fetal immunology, the fetal blood-brain barrier and precise molecular processes during different stages of development will be paramount to continued refinement of in utero therapies. The social and ethical implications of fetal therapy are also of utmost importance, especially in an era of increasing restrictions on reproductive autonomy. This review aims to summarize current efforts toward in utero targeted fetal therapies as well as the history and ethical implications of such endeavors.
期刊介绍:
Seminars in Medical Genetics, Part C of the American Journal of Medical Genetics (AJMG) , serves as both an educational resource and review forum, providing critical, in-depth retrospectives for students, practitioners, and associated professionals working in fields of human and medical genetics. Each issue is guest edited by a researcher in a featured area of genetics, offering a collection of thematic reviews from specialists around the world. Seminars in Medical Genetics publishes four times per year.