Derivation of Diverse Hormone-Releasing Pituitary Cells from Human Pluripotent Stem Cells

Stem Cell Reports. 2016 Jun 14;6(6):858-872. doi: 10.1016/j.stemcr.2016.05.005.

Abstract

Human pluripotent stem cells (hPSCs) provide an unlimited cell source for regenerative medicine. Hormone-producing cells are particularly suitable for cell therapy, and hypopituitarism, a defect in pituitary gland function, represents a promising therapeutic target. Previous studies have derived pituitary lineages from mouse and human ESCs using 3D organoid cultures that mimic the complex events underlying pituitary gland development in vivo. Instead of relying on unknown cellular signals, we present a simple and efficient strategy to derive human pituitary lineages from hPSCs using monolayer culture conditions suitable for cell manufacturing. We demonstrate that purified placode cells can be directed into pituitary fates using defined signals. hPSC-derived pituitary cells show basal and stimulus-induced hormone release in vitro and engraftment and hormone release in vivo after transplantation into a murine model of hypopituitarism. This work lays the foundation for future cell therapy applications in patients with hypopituitarism.

MeSH terms

  • Adrenocorticotropic Hormone / biosynthesis
  • Adrenocorticotropic Hormone / metabolism
  • Animals
  • Benzamides / pharmacology
  • Biomarkers / metabolism
  • Bone Morphogenetic Protein 4 / pharmacology
  • Cell Culture Techniques
  • Cell Differentiation / drug effects
  • Cell- and Tissue-Based Therapy
  • Corticotrophs / cytology
  • Corticotrophs / drug effects
  • Corticotrophs / metabolism*
  • Dioxoles / pharmacology
  • Disease Models, Animal
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / drug effects
  • Embryonic Stem Cells / metabolism*
  • Fibroblast Growth Factors / pharmacology
  • Follicle Stimulating Hormone / biosynthesis
  • Follicle Stimulating Hormone / metabolism
  • GATA3 Transcription Factor / genetics
  • GATA3 Transcription Factor / metabolism
  • Gene Expression
  • Growth Hormone / biosynthesis
  • Growth Hormone / metabolism
  • Homeodomain Proteins / genetics
  • Homeodomain Proteins / metabolism
  • Humans
  • Hypopituitarism / genetics
  • Hypopituitarism / metabolism
  • Hypopituitarism / pathology
  • Hypopituitarism / therapy*
  • Intracellular Signaling Peptides and Proteins / genetics
  • Intracellular Signaling Peptides and Proteins / metabolism
  • Mice
  • Nuclear Proteins / genetics
  • Nuclear Proteins / metabolism
  • Pituitary Gland / metabolism
  • Pituitary Gland / pathology
  • Pluripotent Stem Cells / cytology
  • Pluripotent Stem Cells / drug effects
  • Pluripotent Stem Cells / metabolism*
  • Protein Tyrosine Phosphatases / genetics
  • Protein Tyrosine Phosphatases / metabolism
  • Thyrotrophs / cytology
  • Thyrotrophs / drug effects
  • Thyrotrophs / metabolism*
  • Transcription Factor AP-2 / genetics
  • Transcription Factor AP-2 / metabolism
  • Transcription Factors / genetics
  • Transcription Factors / metabolism

Substances

  • 4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide
  • BMP4 protein, human
  • Benzamides
  • Biomarkers
  • Bone Morphogenetic Protein 4
  • Dioxoles
  • Distal-less homeobox proteins
  • GATA3 Transcription Factor
  • GATA3 protein, human
  • Homeodomain Proteins
  • Intracellular Signaling Peptides and Proteins
  • Nuclear Proteins
  • SIX1 protein, human
  • TFAP2A protein, human
  • Transcription Factor AP-2
  • Transcription Factors
  • Fibroblast Growth Factors
  • Adrenocorticotropic Hormone
  • Follicle Stimulating Hormone
  • Growth Hormone
  • EYA1 protein, human
  • Protein Tyrosine Phosphatases