Pellegrini G, Rama P, Mavilio F, De Luca M
Centre for Regenerative Medicine, Department of Biomedical Sciences, University of Modena and Reggio Emilia, Modena, Italy.
J Pathol. 2009 Jan;217(2):217-28. doi: 10.1002/path.2441.
Regenerative medicine refers to innovative therapies aimed at the permanent restoration of diseased tissues and organs. Regeneration of self-renewing tissues requires specific adult stem cells, which need to be genetically modified to correct inherited genetic diseases. Cultures of epithelial stem cells permanently restore severe skin and mucosal defects, and genetically corrected epidermal stem cells regenerate a normal epidermis in patients carrying junctional epidermolysis bullosa. The keratinocyte stem cell is therefore the only cultured stem cell used both in cell therapy and gene therapy clinical protocols. Epithelial stem cell identification, fate and molecular phenotype have been extensively reviewed, but not in relation to tissue regeneration. In this paper we focus on the localization and molecular characterization of human limbal stem cells in relation to corneal regeneration, and the gene therapy of genetic skin diseases by means of genetically modified epidermal stem cells.
再生医学是指旨在永久性恢复患病组织和器官的创新疗法。自我更新组织的再生需要特定的成体干细胞，这些干细胞需要进行基因改造以纠正遗传性疾病。上皮干细胞培养可永久性修复严重的皮肤和黏膜缺陷，经基因校正的表皮干细胞可使患有交界性大疱性表皮松解症的患者再生出正常表皮。因此，角质形成干细胞是细胞治疗和基因治疗临床方案中唯一使用的培养干细胞。上皮干细胞的鉴定、命运和分子表型已得到广泛综述，但未涉及组织再生方面。在本文中，我们重点关注与角膜再生相关的人角膜缘干细胞的定位和分子特征，以及通过基因改造的表皮干细胞对遗传性皮肤病进行基因治疗。
