Biology, CHDI Foundation, Inc., Suite 100, Los Angeles, CA 90045, USA.
Expert Opin Ther Pat. 2009 Dec;19(12):1639-46. doi: 10.1517/13543770903443105.
The potential of exogenous stem cell or progenitor cell transplantation as a novel therapeutic strategy to address unmet medical needs is a vast and important area of investigation. A recent US patent has been issued to Goldman from the University of Rochester based on pioneering studies with human fetal and adult-derived glial progenitor cells (GPCs), covering the generation of chimeric mouse/human animals.
OBJECTIVE/METHOD: In this patent and associated manuscript, extensive chimerism due to grafting of human GPCs is associated with remyelination and functional rescue of mice congenitally deficient in oligodendrocyte survival and myelination, due to a deletion in the myelin basic protein gene (the shiverer mouse). This review highlights the implications of generating human/mouse chimeric animals for the study of human brain physiology, preclinical studies and the clinical application of progenitor cells towards the development of novel therapeutics for the treatment of demyelinating disorders.
The use of GPCs offers promise for remyelination disorders, and the ability of these cells to repopulate the entire rodent nervous system should allow for the investigation of the physiological properties of human glial derivatives in an in vivo context, enhancing the understanding of mechanisms with a primary effect through the modulation of human glial cell biology.
外源性干细胞或祖细胞移植作为一种解决未满足医疗需求的新治疗策略具有广阔而重要的研究价值。罗彻斯特大学的 Goldman 获得了一项美国专利，该专利基于对人胎儿和成体来源的神经胶质前体细胞（GPC）的开创性研究，涵盖了嵌合鼠/人动物的产生。
目的/方法：在这项专利和相关论文中，由于移植人 GPC 导致的广泛嵌合与髓鞘再生和功能恢复有关，这些小鼠由于髓鞘碱性蛋白基因（shiverer 小鼠）缺失而先天缺乏少突胶质细胞存活和髓鞘形成。本综述强调了生成人/鼠嵌合动物对于研究人类大脑生理学、临床前研究以及祖细胞在开发治疗脱髓鞘疾病的新型治疗方法中的临床应用的意义。
GPC 的使用为髓鞘再生障碍提供了希望，这些细胞能够重新填充整个啮齿动物神经系统，这应该允许在体内环境中研究人类神经胶质衍生物的生理特性，通过调节人类神经胶质细胞生物学来增强对具有主要影响的机制的理解。
