Human Pluripotent-Derived Lineages for Repairing Hypopituitarism
Human pluripotent stem cells (hPSCs) present a potentially unlimited source of specialized cell types for regenerative medicine. Over the last few years there has been rapid progress in realizing this potential by developing protocols to generate disease-
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Abstract Human pluripotent stem cells (hPSCs) present a potentially unlimited source of specialized cell types for regenerative medicine. Over the last few years there has been rapid progress in realizing this potential by developing protocols to generate disease-relevant cell types in vitro on demand. The approach was particularly successful for the nervous system, where the field is at the verge of human translation for several indications, including the treatment of eye disorders, Parkinson’s disease and spinal cord injury. More recently, there has also been success in deriving anterior pituitary lineages from both mouse and human pluripotent stem cells. In vitro-derived pituitary hormone-producing cell types present an attractive source for repair in patients with hypopituitarism. However, several hurdles remain towards realizing this goal. In particular, there is a need to further improve the efficiency and precision with which specific hormone-producing lineages can be derived. Furthermore, it will be important to assess the potential of both ectopic and orthotopic transplantation strategies to achieve meaningful hormone replacement. The ultimate challenge will be repair that moves beyond hormone replacement towards the full functional integration of the grafted cells into the complex regulatory endocrine network controlled by the human pituitary gland.
L. Studer (*) Developmental Biology, The Center for Stem Cell Biology, Memorial Sloan-Kettering Cancer Center, New York, NY, USA e-mail: [email protected] V. Tabar (*) Department of Neurosurgery, The Center for Stem Cell Biology, Memorial Sloan-Kettering Cancer Center, New York, NY, USA e-mail: [email protected] © The Author(s) 2016 D. Pfaff, Y. Christen (eds.), Stem Cells in Neuroendocrinology, Research and Perspectives in Endocrine Interactions, DOI 10.1007/978-3-319-41603-8_3
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L. Studer and V. Tabar
Derivation of Human Neural Cell Types for Regenerative Medicine The isolation of human embryonic stem cells (ESCs; Thomson et al. 1998) and the remarkable feat of reprogramming somatic cells back to pluripotency via induced pluripotent stem cell (iPSC) technology (Takahashi et al. 2007; Takahashi and Yamanaka 2006; Yu et al. 2007) have set the stage for a new era of regenerative medicine. Human pluripotent stem cells (hPSCs), a term comprising both human ESCs and iPSCs, are characterized by their potential to differentiate into any cell lineage of the body. For many years, the main challenge in the field has been to capture the broad differentiation potential of hPSCs towards specific cell lineages relevant to modeling and treating human disease. However, there has been considerable progress recently in establishing differentiation protocol for many key lineages such as endoderm-derived insulin-producing pancreatic cells (Pagliuca et al. 2014; Rezania et al. 2014) for the treatment of diabetes or mesoderm-derived cardiac cells for heart repair (Chong et al. 2014). Some of the most dramatic successes, however, have involved ectoderm-derived lineages, in
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