基因向造血干细胞的转移：对人类疾病基因治疗的意义。
Gene transfer to hematopoietic stem cells: implications for gene therapy of human disease.
作者信息
Dunbar C E
机构信息
Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, Maryland 20892, USA.
出版信息
Annu Rev Med. 1996;47:11-20. doi: 10.1146/annurev.med.47.1.11.
PMID:8712765
Abstract
Transfer of new genetic material to hematopoietic stem cells and expression of the gene product in daughter cells of various lineages is an exciting approach to the treatment of congenital and acquired human diseases. This review summarizes the current status of retroviral and adeno-associated viral vectors for gene transfer to human hematopoietic cells, including extensive preclinical data as well as preliminary results from ongoing clinical trials.
摘要
将新的遗传物质转移到造血干细胞，并使基因产物在各种谱系的子代细胞中表达，是治疗先天性和后天性人类疾病的一种令人兴奋的方法。本综述总结了用于将基因转移到人类造血细胞的逆转录病毒和腺相关病毒载体的现状，包括广泛的临床前数据以及正在进行的临床试验的初步结果。
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