Over the past decade, amniotic fluid-derived stem cells have emerged as a novel experimental approach aimed at improving outcomes in children with congenital anomalies, including spina bifida, heart defects, and diaphragmatic hernia. Interest in these cells for the treatment of prenatally diagnosed diseases has arisen based on numerous studies demonstrating the relative ease of harvesting an abundant quantity of amniocytes from a small aliquot of fluid, the unique properties of amniocytes themselves, and the beneficial effects of amniotic fluid-derived stem cells in experimental animal models. This report gives a brief overview of the rationale and current status of amniotic fluid stem cell-based therapies, focusing on its relevance to birth defects affecting the fetus and neonate. The author proposes a roadmap for further study that would be required prior to clinical application of amniotic fluid stem cell technologies. Stem Cells Translational Medicine 2018;7:767–773.
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