Heuckeroth RO (2018) Hirschsprung disease–integrating basic science and clinical medicine to improve outcomes. Nat Rev Gastroenterol Hepatol 15:152–167. https://doi.org/10.1038/nrgastro.2017.149
Burns AJ, Goldstein AM, Newgreen DF, Stamp L, Schäfer KH, Metzger M, Hotta R, Young HM, Andrews PW, Thapar N, Belkind-Gerson J, Bondurand N, Bornstein JC, Chan WY, Cheah K, Gershon MD, Heuckeroth RO, Hofstra RM, Just L, Kapur RP, King SK, McCann CJ, Nagy N, Ngan E, Obermayr F, Pachnis V, Pasricha PJ, Sham MH, Tam P, Vanden Berghe P (2016) White paper on guidelines concerning enteric nervous system stem cell therapy for enteric neuropathies. Dev Biol 417:229–251. https://doi.org/10.1016/j.ydbio.2016.04.001
Article PubMed PubMed Central Google Scholar
Goldstein AM, Thapar N, Karunaratne TB, De Giorgio R (2016) Clinical aspects of neurointestinal disease: pathophysiology, diagnosis, and treatment. Dev Biol 417:217–228. https://doi.org/10.1016/j.ydbio.2016.03.032
Westfal ML, Goldstein AM (2017) Pediatric enteric neuropathies: diagnosis and current management. Curr Opin Pediatr 29:347–353. https://doi.org/10.1097/mop.0000000000000486
Article PubMed PubMed Central Google Scholar
Zimmer J, Tomuschat C, Puri P (2016) Long-term results of transanal pull-through for Hirschsprung’s disease: a meta-analysis. Pediatr Surg Int 32:743–749. https://doi.org/10.1007/s00383-016-3908-z
Laughlin DM, Friedmacher F, Puri P (2012) Total colonic aganglionosis: a systematic review and meta-analysis of long-term clinical outcome. Pediatr Surg Int 28:773–779. https://doi.org/10.1007/s00383-012-3117-3
Alhawaj AF (2022) Stem cell-based therapy for Hirschsprung disease, do we have the guts to treat? Gene Ther 29:578–587. https://doi.org/10.1038/s41434-021-00268-4
Trounson A, McDonald C (2015) Stem cell therapies in clinical trials: progress and challenges. Cell Stem Cell 17:11–22. https://doi.org/10.1016/j.stem.2015.06.007
McCann CJ, Borrelli O, Thapar N (2018) Stem cell therapy in severe pediatric motility disorders. Curr Opin Pharmacol 43:145–149. https://doi.org/10.1016/j.coph.2018.09.004
Metzger M, Bareiss PM, Danker T, Wagner S, Hennenlotter J, Guenther E, Obermayr F, Stenzl A, Koenigsrainer A, Skutella T, Just L (2009) Expansion and differentiation of neural progenitors derived from the human adult enteric nervous system. Gastroenterology 137:2063-2073.e2064. https://doi.org/10.1053/j.gastro.2009.06.038
Metzger M, Caldwell C, Barlow AJ, Burns AJ, Thapar N (2009) Enteric nervous system stem cells derived from human gut mucosa for the treatment of aganglionic gut disorders. Gastroenterology 136:2214–2225. https://doi.org/10.1053/j.gastro.2009.02.048
Hotta R, Natarajan D, Thapar N (2009) Potential of cell therapy to treat pediatric motility disorders. Semin Pediatr Surg 18:263–273. https://doi.org/10.1053/j.sempedsurg.2009.07.008
Micci MA, Pasricha PJ (2007) Neural stem cells for the treatment of disorders of the enteric nervous system: strategies and challenges. Dev Dyn 236:33–43. https://doi.org/10.1002/dvdy.20975
Laranjeira C, Pachnis V (2009) Enteric nervous system development: recent progress and future challenges. Auton Neurosci 151:61–69. https://doi.org/10.1016/j.autneu.2009.09.001
Sasselli V, Pachnis V, Burns AJ (2012) The enteric nervous system. Dev Biol 366:64–73. https://doi.org/10.1016/j.ydbio.2012.01.012
Taraviras S, Marcos-Gutierrez CV, Durbec P, Jani H, Grigoriou M, Sukumaran M, Wang LC, Hynes M, Raisman G, Pachnis V (1999) Signalling by the RET receptor tyrosine kinase and its role in the development of the mammalian enteric nervous system. Development 126:2785–2797. https://doi.org/10.1242/dev.126.12.2785
Airaksinen MS, Saarma M (2002) The GDNF family: signalling, biological functions and therapeutic value. Nat Rev Neurosci 3:383–394. https://doi.org/10.1038/nrn812
Young HM, Hearn CJ, Farlie PG, Canty AJ, Thomas PQ, Newgreen DF (2001) GDNF is a chemoattractant for enteric neural cells. Dev Biol 229:503–516. https://doi.org/10.1006/dbio.2000.0100
Natarajan D, Marcos-Gutierrez C, Pachnis V, de Graaff E (2002) Requirement of signalling by receptor tyrosine kinase RET for the directed migration of enteric nervous system progenitor cells during mammalian embryogenesis. Development 129:5151–5160. https://doi.org/10.1242/dev.129.22.5151
McKeown SJ, Mohsenipour M, Bergner AJ, Young HM, Stamp LA (2017) Exposure to GDNF enhances the ability of enteric neural progenitors to generate an enteric nervous system. Stem Cell Reports 8:476–488. https://doi.org/10.1016/j.stemcr.2016.12.013
Article PubMed PubMed Central Google Scholar
Heanue TA, Pachnis V (2007) Enteric nervous system development and Hirschsprung’s disease: advances in genetic and stem cell studies. Nat Rev Neurosci 8:466–479. https://doi.org/10.1038/nrn2137
Shibata S, Yasuda A, Renault-Mihara F, Suyama S, Katoh H, Inoue T, Inoue YU, Nagoshi N, Sato M, Nakamura M, Akazawa C, Okano H (2010) Sox10-Venus mice: a new tool for real-time labeling of neural crest lineage cells and oligodendrocytes. Mol Brain 3:31. https://doi.org/10.1186/1756-6606-3-31
Article PubMed PubMed Central Google Scholar
Schriemer D, Sribudiani Y, IJpma A, Natarajan D, MacKenzie KC, Metzger M, Binder E, Burns AJ, Thapar N, Hofstra RMW, Eggen BJL (2016) Regulators of gene expression in enteric neural crest cells are putative Hirschsprung disease genes. Dev Biol 416:255–265. https://doi.org/10.1016/j.ydbio.2016.06.004
Baloh RH, Enomoto H, Johnson EM Jr, Milbrandt J (2000) The GDNF family ligands and receptors–implications for neural development. Curr Opin Neurobiol 10:103–110. https://doi.org/10.1016/s0959-4388(99)00048-3
Moore MW, Klein RD, Fariñas I, Sauer H, Armanini M, Phillips H, Reichardt LF, Ryan AM, Carver-Moore K, Rosenthal A (1996) Renal and neuronal abnormalities in mice lacking GDNF. Nature 382:76–79. https://doi.org/10.1038/382076a0
Airaksinen MS, Titievsky A, Saarma M (1999) GDNF family neurotrophic factor signaling: four masters, one servant? Mol Cell Neurosci 13:313–325. https://doi.org/10.1006/mcne.1999.0754
Enomoto H, Crawford PA, Gorodinsky A, Heuckeroth RO, Johnson EM Jr, Milbrandt J (2001) RET signaling is essential for migration, axonal growth and axon guidance of developing sympathetic neurons. Development 128:3963–3974. https://doi.org/10.1242/dev.128.20.3963
Schuchardt A, D’Agati V, Larsson-Blomberg L, Costantini F, Pachnis V (1994) Defects in the kidney and enteric nervous system of mice lacking the tyrosine kinase receptor Ret. Nature 367:380–383. https://doi.org/10.1038/367380a0
Ngan ES, Shum CK, Poon HC, Sham MH, Garcia-Barcelo MM, Lui VC, Tam PK (2008) Prokineticin-1 (Prok-1) works coordinately with glial cell line-derived neurotrophic factor (GDNF) to mediate proliferation and differentiation of enteric neural crest cells. Biochim Biophys Acta 1783:467–478. https://doi.org/10.1016/j.bbamcr.2007.09.005
Anitha M, Chandrasekharan B, Salgado JR, Grouzmann E, Mwangi S, Sitaraman SV, Srinivasan S (2006) Glial-derived neurotrophic factor modulates enteric neuronal survival and proliferation through neuropeptide Y. Gastroenterology 131:1164–1178. https://doi.org/10.1053/j.gastro.2006.07.019
Barlow AJ, Wallace AS, Thapar N, Burns AJ (2008) Critical numbers of neural crest cells are required in the pathways from the neural tube to the foregut to ensure complete enteric nervous system formation. Development 135:1681–1691. https:
Comments (0)