Stem cell therapy in severe pediatric motility disorders.

Highlights • Currently, very limited treatments and poor outcomes for enteric neuropathies. • Neural stem cell-derived therapies provide the prospect of curative treatments. • Enteric neural stem cells harvested from postnatal human gastrointestinal tract. • Induced pluripotent stem cells can be directed towards an enteric neural fate. • Transplanted neural stem cells rescue function in enteric neuropathy animal models. Pediatric gastrointestinal motility disorders represent a range of severe developmental or acquired conditions that disrupt enteric neuromuscular function. Current medical and surgical therapeutic options are very limited but recent advances have highlighted the possibility of improved or curative stem cell-based treatments. Not only has the ability to harvest, propagate and transplant human-derived enteric neural stem cells (ENSCs) been demonstrated but recent in vivo transplantation studies have confirmed that ENSCs are capable of engraftment within recipient intestine of animal models of enteric neuropathy and effecting functional rescue. Pluripotent stem cell-derived cells and pharmacological modulation of both endogenous and transplanted neural stem cells have further enhanced the exciting prospect of clinical application of such stem cell-based therapies in the near future. [ABSTRACT FROM AUTHOR]