The Current State and Future of Fetal Therapies

Abstract

Through next-generation sequencing, we can now detect a myriad of rare genetic diseases in utero that were previously not diagnosed until after birth. Fetal therapies hold strong promise for transforming prenatal management of genetic diseases, preventing adverse effects from organ damage in utero, and improving the grim perinatal outcomes of numerous genetic diseases. Many novel, in utero therapies are under investigation for genetic diseases using hematopoietic stem cells, cellular pathway inhibitors, viral vectors, and other biotechnologies. This article reviews emerging fetal therapies, as well as existing guidance for their development, considerations for their safety, and ethical and societal implications.