Pediatric Neurogastroenterology

Part of the series Clinical Gastroenterology pp 493-505


Cellular-Based Therapies for Pediatric GI Motility Disorders

  • Ryo HottaAffiliated withDepartment of Anatomy and Neuroscience, The University of MelbourneDepartment of Pediatric Surgery, Keio University School of Medicine
  • , Dipa NatarajanAffiliated withNeural Development Unit, UCL, Institute of Child Health
  • , Alan J. BurnsAffiliated withNeural Development Unit, UCL, Institute of Child Health
  • , Nikhil ThaparAffiliated withGastroenterology Unit, Division of Neurogastroenterology and Motility, University College London, Institute of Child HealthDepartment of Paediatric Gastroenterology, Great Ormond Street Hospital for Children Email author 

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The therapeutic options for many gastrointestinal motility disorders remain inadequate and limited to palliative interventions. Recent advances in molecular biology and genetics have led to the identification of stem cells as potential tools for curative therapies. The field of neural stem cell therapies for enteric neuropathies has seen significant progress in recent years. A variety of sources for such neural stem cells have been identified ranging from embryonic stem cells to those derived from the CNS and, perhaps of most interest, from the gastrointestinal tract itself. The latter have been harvested from postnatal human gut even using minimally invasive techniques such as conventional endoscopy raising exciting possibilities for therapy including autologous therapy. A number of key challenges remain, however, before effective clinical application. These include better understanding of target diseases, the need to tailor cellular tools to optimize the treatment of individual diseases, and effective assessment of transplant success. Exciting developments including the identification of new stem cell sources such as induced pluripotent stem cells and improved access to donor tissue using less invasive techniques raise further hope for stem cells as effective therapies for enteric neuromuscular diseases.