Burns Alan J, Goldstein Allan M, Newgreen Donald F, Stamp Lincon, Schäfer Karl-Herbert, Metzger Marco, Hotta Ryo, Young Heather M, Andrews Peter W, Thapar Nikhil, Belkind-Gerson Jaime, Bondurand Nadege, Bornstein Joel C, Chan Wood Yee, Cheah Kathryn, Gershon Michael D, Heuckeroth Robert O, Hofstra Robert M W, Just Lothar, Kapur Raj P, King Sebastian K, McCann Conor J, Nagy Nandor, Ngan Elly, Obermayr Florian, Pachnis Vassilis, Pasricha Pankaj J, Sham Mai Har, Tam Paul, Vanden Berghe Pieter
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.
Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Dev Biol. 2016 Sep 15;417(2):229-51. doi: 10.1016/j.ydbio.2016.04.001. Epub 2016 Apr 5.
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.
在过去20年里,人们越来越关注开发基于新型干细胞的疗法,用于治疗影响胃肠道肠神经系统(ENS)的疾病(即所谓的肠神经病变)。这里的想法是,可以将ENS祖细胞/干细胞移植到肠壁中,以替代ENS中受损或缺失的神经元和神经胶质细胞。本白皮书阐述了专家们对于识别、分离、纯化、扩增和优化ENS干细胞、将其移植到肠道以及评估移植成功率(包括肠道功能恢复情况)的常用方法和途径的看法。我们还强调了要从动物模型的成功临床前研究推进到临床ENS干细胞疗法必须克服的障碍。