Directed differentiation of human pluripotent stem cells (hPSCs) enables the derivation of distinct functional cell types with great promise for disease modeling and regenerative medicine. Our research has been focused on instructing hPSCs into acquiring highly specific identities of the peripheral nervous system (PNS) cell types. Access to these cells at large scale and with high precision allows us to systematically dissect developmental and disease mechanisms and leverage their potential for drug discovery and cell therapy.
The PNS is developed from the Neural crest (NC). NC are migratory multipotent progenitors that delaminates from the developing neural tube and give rise to a diverse array of cell types in different organs. NC are transient embryonic populations and therefore difficult to obtain from primary sources, particularly in humans. Derivation of NC from hPSCs offers an alternative way to access the precursors of the peripheral nervous system for developmental and disease related studies. These strategies set the foundation for increasing the resolution of patterning and directed derivation of specific PNS components.
The key components of the PNS are sensory, autonomic and enteric neurons and the specialized glia associated with them. The ability to access human PNS cell types with unprecedented precision and scale and employing these cells for disease modeling, drug discovery and cells therapy, provides a unique and exciting opportunity to address long standing questions in the field with the ultimate goal of designing novel strategies for treatment of peripheral neuropathies.