Genzyme Corporation, Science Center, Framingham, MA 01701, USA.
Neuropharmacology. 2010 May;58(6):845-54. doi: 10.1016/j.neuropharm.2009.12.015. Epub 2009 Dec 28.
Over the last decade, the potential for therapeutic use of stem cell transplantation for cell replacement or as cellular vectors for gene delivery for neurometabolic and neurodegenerative diseases has received a great deal of interest. There has been substantial progress in our understanding of stem cell biology. Potential applications of cell-mediated therapy include direct cell replacement or protection and repair of the host nervous system. Given the complexities of the cellular organization of the nervous system, especially in diseased states, it seems that using stem cells as cellular vectors to prevent or ameliorate neurological disorders rather than cell replacement and the regrowth of damaged circuitry is more likely to succeed in the near term. Recent success in the treatment of lysosomal storage diseases with genetically modified stem cells support this notion. In Alzheimer's and Parkinson's diseases, stem cell therapy is at its early stages and data generated in animal models and clinical trials using other cell types suggest that a combination of gene and stem cell therapy may be an optimal therapeutic paradigm.
在过去的十年中，干细胞移植在神经代谢和神经退行性疾病中作为细胞替代物或作为基因传递的细胞载体的治疗用途引起了广泛关注。人们对干细胞生物学的理解已经取得了实质性的进展。细胞介导治疗的潜在应用包括直接细胞替代或宿主神经系统的保护和修复。鉴于神经系统的细胞组织的复杂性，尤其是在患病状态下，使用干细胞作为细胞载体来预防或改善神经紊乱而不是细胞替代和受损电路的再生似乎更有可能在短期内取得成功。最近使用基因修饰的干细胞治疗溶酶体贮积症的成功支持了这一观点。在阿尔茨海默病和帕金森病中，干细胞治疗还处于早期阶段，并且在使用其他细胞类型的动物模型和临床试验中生成的数据表明，基因和干细胞治疗的组合可能是一种最佳的治疗范例。
