title: White paper on guidelines concerning enteric nervous system stem cell therapy for enteric neuropathies.
abstract: Over the last 20 years, there has been increasing focus on the development of novel stem cell based therapies for the treatment of disorders and diseases affecting the enteric nervous system (ENS) of the gastrointestinal tract (so-called enteric neuropathies). Here, the idea is that ENS progenitor/stem cells could be transplanted into the gut wall to replace the damaged or absent neurons and glia of the ENS. This White Paper sets out experts' views on the commonly used methods and approaches to identify, isolate, purify, expand and optimize ENS stem cells, transplant them into the bowel, and assess transplant success, including restoration of gut function. We also highlight obstacles that must be overcome in order to progress from successful preclinical studies in animal models to ENS stem cell therapies in the clinic.
authors.affiliation: Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Clinical Genetics, Erasmus Medical Center, Rotterdam, The Netherlands. Electronic address: alan.burns@ucl.ac.uk.
authors.forename: Alan J
authors.initials: AJ
authors.lastname: Burns
authors.affiliation: Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
authors.forename: Allan M
authors.initials: AM
authors.lastname: Goldstein
authors.affiliation: Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville 3052, Victoria, Australia.
authors.forename: Donald F
authors.initials: DF
authors.lastname: Newgreen
authors.affiliation: Department of Anatomy and Neuroscience, University of Melbourne, Parkville, Victoria 3010, Australia.
authors.forename: Lincon
authors.initials: L
authors.lastname: Stamp
authors.affiliation: University of Applied Sciences, Kaiserlautern, Germany; Clinic of Pediatric Surgery, University Hospital Mannheim, University Heidelberg, Germany.
authors.forename: Karl-Herbert
authors.initials: KH
authors.lastname: Schäfer
authors.affiliation: Fraunhofer-Institute Interfacial Engineering and Biotechnology IGB Translational Centre - Würzburg branch and University Hospital Würzburg - Tissue Engineering and Regenerative Medicine (TERM), Würzburg, Germany.
authors.forename: Marco
authors.initials: M
authors.lastname: Metzger
authors.affiliation: Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
authors.forename: Ryo
authors.initials: R
authors.lastname: Hotta
authors.affiliation: Department of Anatomy and Neuroscience, University of Melbourne, Parkville, Victoria 3010, Australia.
authors.forename: Heather M
authors.initials: HM
authors.lastname: Young
authors.affiliation: Centre for Stem Cell Biology, Department of Biomedical Science, University of Sheffield, Sheffield, UK.
authors.forename: Peter W
authors.initials: PW
authors.lastname: Andrews
authors.affiliation: Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK.
authors.forename: Nikhil
authors.initials: N
authors.lastname: Thapar
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Massachusetts General Hospital for Children, Harvard Medical School, Boston, USA.
authors.forename: Jaime
authors.initials: J
authors.lastname: Belkind-Gerson
authors.affiliation: INSERM U955, 51 Avenue du Maréchal de Lattre de Tassigny, F-94000 Créteil, France; Université Paris-Est, UPEC, F-94000 Créteil, France.
authors.forename: Nadege
authors.initials: N
authors.lastname: Bondurand
authors.affiliation: Department of Physiology, University of Melbourne, Parkville, Victoria 3010, Australia.
authors.forename: Joel C
authors.initials: JC
authors.lastname: Bornstein
authors.affiliation: School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong.
authors.forename: Wood Yee
authors.initials: WY
authors.lastname: Chan
authors.affiliation: School of Biomedical Sciences, The University of Hong Kong, Hong Kong.
authors.forename: Kathryn
authors.initials: K
authors.lastname: Cheah
authors.affiliation: Department of Pathology and Cell Biology, Columbia University, New York 10032, USA.
authors.forename: Michael D
authors.initials: MD
authors.lastname: Gershon
authors.affiliation: Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA 19104, USA; Perelman School of Medicine at the University of Pennsylvania, Abramson Research Center, Philadelphia, PA 19104, USA.
authors.forename: Robert O
authors.initials: RO
authors.lastname: Heuckeroth
authors.affiliation: Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Clinical Genetics, Erasmus Medical Center, Rotterdam, The Netherlands.
authors.forename: Robert M W
authors.initials: RM
authors.lastname: Hofstra
authors.affiliation: Institute of Clinical Anatomy and Cell Analysis, University of Tübingen, Germany.
authors.forename: Lothar
authors.initials: L
authors.lastname: Just
authors.affiliation: Department of Pathology, University of Washington and Seattle Children's Hospital, Seattle, WA, USA.
authors.forename: Raj P
authors.initials: RP
authors.lastname: Kapur
authors.affiliation: Department of Paediatric and Neonatal Surgery, The Royal Children's Hospital, Melbourne, Australia.
authors.forename: Sebastian K
authors.initials: SK
authors.lastname: King
authors.affiliation: Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK.
authors.forename: Conor J
authors.initials: CJ
authors.lastname: McCann
authors.affiliation: Department of Anatomy, Histology and Embryology, Faculty of Medicine, Semmelweis University, Budapest, Hungary.
authors.forename: Nandor
authors.initials: N
authors.lastname: Nagy
authors.affiliation: Department of Surgery, The University of Hong Kong, Hong Kong.
authors.forename: Elly
authors.initials: E
authors.lastname: Ngan
authors.affiliation: Department of Pediatric Surgery and Pediatric Urology, University Children's Hospital Tübingen, D-72076 Tübingen, Germany.
authors.forename: Florian
authors.initials: F
authors.lastname: Obermayr
authors.affiliation: The Francis Crick Institute, Mill Hill Laboratory, London, UK.
authors.forename: Vassilis
authors.initials: V
authors.lastname: Pachnis
authors.affiliation: Johns Hopkins Center for Neurogastroenterology, Baltimore, MD, USA.
authors.forename: Pankaj J
authors.initials: PJ
authors.lastname: Pasricha
authors.affiliation: Department of Biochemistry, The University of Hong Kong, Hong Kong.
authors.forename: Mai Har
authors.initials: MH
authors.lastname: Sham
authors.affiliation: Department of Surgery, The University of Hong Kong, Hong Kong.
authors.forename: Paul
authors.initials: P
authors.lastname: Tam
authors.affiliation: Laboratory for Enteric NeuroScience (LENS), TARGID, University of Leuven, Belgium.
authors.forename: Pieter
authors.initials: P
authors.lastname: Vanden Berghe
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D004195:Disease Models, Animal; D017615:Enteric Nervous System / Q000473:pathology*; D041981:Gastrointestinal Tract / Q000294:innervation / Q000473:pathology*; D017408:Guidelines as Topic; D006627:Hirschsprung Disease / Q000473:pathology / Q000628:therapy*; D006801:Humans; D007418:Intestinal Pseudo-Obstruction / Q000473:pathology / Q000628:therapy*; D058953:Neural Stem Cells / Q000637:transplantation*; D033581:Stem Cell Transplantation
keywords: Cell replacement therapy; Enteric nervous system; Enteric neuropathies; Hirschsprung disease; Stem cells