In Utero Therapies, the Next Frontier.

IF 4.4 3区 医学 Q2 GENETICS & HEREDITY American Journal of Medical Genetics Part C: Seminars in Medical Genetics Pub Date : 2025-09-01 Epub Date: 2025-03-24 DOI:10.1002/ajmg.c.32135
Teodora R Kolarova, Edith Cheng
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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.

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子宫内治疗,下一个前沿。
在过去的十年中,分子诊断的进步和对多种遗传条件下病理生理过程的广泛理解导致了许多靶向治疗的设计和实施。在产前领域,这些进步迅速改变了产前诊断领域,并引起了人们对旨在纠正子宫内特定疾病机制的干预措施的热情,以防止不可逆的损伤和/或利用胎儿生理学来提高这些治疗的有效性。尽管许多有希望的试验正在进行中,但继续努力充分阐明胎儿免疫学的复杂性,胎儿血脑屏障和不同发育阶段的精确分子过程将是继续完善子宫内治疗的首要任务。胎儿治疗的社会和伦理影响也至关重要,特别是在一个对生殖自主权限制日益增加的时代。这篇综述的目的是总结目前对子宫内靶向胎儿治疗的努力,以及这些努力的历史和伦理意义。
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来源期刊
CiteScore
7.00
自引率
0.00%
发文量
42
审稿时长
>12 weeks
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.
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