Kohn D B
University of Southern California School of Medicine, Los Angeles, USA.
Curr Opin Pediatr. 1995 Feb;7(1):56-63. doi: 10.1097/00008480-199502000-00011.
Gene transfer into hematopoietic stem cells may provide a novel treatment for a number of congenital hematologic and immunologic diseases. Retroviral vectors are currently the most effective gene transfer system, but they have significant limitations. New approaches using other vector systems, stem cells from umbilical cord blood, or cytokine-mobilized peripheral blood stem cells may increase gene transfer efficiency. The current capabilities and obstacles to successful implementation of gene therapy with hematopoietic stem cells are discussed.
将基因导入造血干细胞可能为多种先天性血液学和免疫学疾病提供一种新的治疗方法。逆转录病毒载体是目前最有效的基因转移系统，但它们有显著的局限性。使用其他载体系统、脐带血干细胞或细胞因子动员的外周血干细胞的新方法可能会提高基因转移效率。本文讨论了目前利用造血干细胞成功实施基因治疗的能力和障碍。
