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Semin Fetal Neonatal Med. 2010 Feb;15(1):46-51. doi: 10.1016/j.siny.2009.05.005. Epub 2009 Jun 21.

Stem cell and genetic therapies for the fetus.

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  • 1Children's Center for Fetal Research, Children's Hospital of Philadelphia, PA, USA.


Advances in prenatal diagnosis have led to the prenatal management of a variety of congenital diseases. Although prenatal stem cell and gene therapy await clinical application, they offer tremendous potential for the treatment of many genetic disorders. Normal developmental events in the fetus offer unique biologic advantages for the engraftment of hematopoietic stem cells and efficient gene transfer that are not present after birth. Although barriers to hematopoietic stem cell engraftment exist, progress has been made and preclinical studies are now underway for strategies based on prenatal tolerance induction to facilitate postnatal cellular transplantation. Similarly, in-utero gene therapy shows experimental promise for a host of diseases and proof-in-principle has been demonstrated in murine models, but ethical and safety issues still need to be addressed. Here we review the current status and future potential of prenatal cellular and genetic therapy.

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