Table 1.

Application	Examples
In vitro cell derivation for humanized model systems	Neurons Amyotrophic lateral sclerosis (11) Rett syndrome (33) Familial dysautonomia (28)
	Cardiomyocytes Long QT syndrome (21,35)
Drug development studies	Cardiomyocytes: Long QT syndrome (21)
Derivation of cells from compartments not readily accessible or amenable to culture	Lung cells (48) Epithelial cells Endothelial cells Smooth muscle cells
Studies of epigenetic variants, and other modifiers of cell phenotypes	Epigenetic modification of gene expression and cell phenotype (38)
In vitro and ex vivo tissue engineering to reconstruct tissues for study and/or therapy	Bioartificial lung development for study and/or autologous transplantation (39, 42) Bioartificial heart development for study and/or autologous transplantation
Cell-based therapies	No human studies to date Potential to support current autologous lung-specific cell-based therapies, such as Pulmonary Hypertension: Assessment of Cell Therapy Trial