Program in Stem Cell & Regenerative Biology, Sanford-Burnham Medical Research Institute, La Jolla, CA 92037, USA.
Regen Med. 2011 Nov;6(6 Suppl):25-30. doi: 10.2217/rme.11.85.
The prospect of using neural cells derived from stem cells or from reprogrammed adult somatic cells provides a unique opportunity in cell therapy and drug discovery for developing novel strategies for brain repair. Cell-based therapeutic approaches for treating CNS afflictions caused by disease or injury aim to promote structural repair of the injured or diseased neural tissue, an outcome currently not achieved by drug therapy. Preclinical research in animal models of various diseases or injuries report that grafts of neural cells enhance endogenous repair, provide neurotrophic support to neurons undergoing degeneration and replace lost neural cells. In recent years, the sources of neural cells for treating neurological disorders have been rapidly expanding and in addition to offering therapeutic potential, neural cell products hold promise for disease modeling and drug discovery use. Specific neural cell types have been derived from adult or fetal brain, from human embryonic stem cells, from induced pluripotent stem cells and directly transdifferentiated from adult somatic cells, such as skin cells. It is yet to be determined if the latter approach will evolve into a paradigm shift in the fields of stem cell research and regenerative medicine. These multiple sources of neural cells cover a wide spectrum of safety that needs to be balanced with efficacy to determine the viability of the cellular product. In this article, we will review novel sources of neural cells and discuss current obstacles to developing them into viable cellular products for treating neurological disorders.
利用干细胞或重编程的成体细胞衍生的神经细胞，为开发用于大脑修复的新型策略提供了细胞治疗和药物发现方面的独特机会。基于细胞的治疗方法旨在促进疾病或损伤引起的中枢神经系统损伤的结构修复，而目前药物治疗尚无法实现这一目标。各种疾病或损伤的动物模型的临床前研究报告称，神经细胞移植可增强内源性修复、为正在退化的神经元提供神经营养支持并替代丢失的神经细胞。近年来，用于治疗神经紊乱的神经细胞来源正在迅速扩大，除了具有治疗潜力外，神经细胞产品还有望用于疾病建模和药物发现。特定的神经细胞类型已经从成人或胎儿大脑、人胚胎干细胞、诱导多能干细胞和直接从成体细胞（如皮肤细胞）中转分化而来。尚不确定后者是否会成为干细胞研究和再生医学领域的范式转变。这些多种神经细胞来源涵盖了广泛的安全性范围，需要与疗效相平衡，以确定细胞产品的可行性。在本文中，我们将回顾神经细胞的新来源，并讨论将其开发为用于治疗神经紊乱的可行细胞产品所面临的当前障碍。
