Abstract

The amniotic fluid (AF) provides a source of stem cells with potential use for in utero cell gene therapy to treat congenital disease for the following reasons: (1) amniotic fluid stem cells (AFSCs) are broadly multipotent with high self-renewal capacity, low antigenicity that are able to differentiate into lineages of all three germ layers but are not tumorigenic when injected in vivo; (2) collection of AF can be performed with patient consent at various stages of pregnancy such as amniocentesis, amnio-drainage, or during caesarean delivery, giving a wide range of gestational ages; (3) a variety of stem cell populations are available, which can differentiate down hematopoietic and mesenchymal lineages. Expansion of mesenchymal derived–AFSCs is straightforward, and biobanks are becoming available. Maintaining hematopoiesis after AFSC expansion is more challenging. In this chapter we will discuss how AFSCs could be used in allogeneic or autologous transplantation to treat congenital disease in utero.

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