遗传增强
病毒载体
干细胞
造血
转基因
生物
基因治疗载体
载体(分子生物学)
基因传递
造血干细胞
转导(生物物理学)
慢病毒
病毒学
癌症研究
基因
细胞生物学
病毒
遗传学
重组DNA
病毒性疾病
生物化学
作者
Niels-Bjarne Woods,Andreas Ooka,Stefan Karlsson
出处
期刊:Leukemia
[Springer Nature]
日期:2002-04-01
卷期号:16 (4): 563-569
被引量:38
标识
DOI:10.1038/sj.leu.2402447
摘要
Lentiviral vectors are promising tools for the development of gene therapy since they can transduce both quiescent and dividing target cells. Lentiviral vectors may be particularly promising gene delivery tools for hematopoietic stem cells since these target cells tend to be quiescent and are therefore difficult target cells for vectors that require dividing targets. Human hematopoietic stem cells that can repopulate NOD/SCID mice have been efficiently transduced using HIV-1-based lentiviral vectors and similar vectors can also transduce murine hematopoietic stem cells. HIV-1 vectors that contain strong general promoters can generate high levels of transgene expression and very high expression levels can be generated in erythroid cells in vivo using beta-globin regulatory sequences to control the expression of the transgene. Current lentiviral vectors have a similar level of biosafety as oncoretroviral vectors and can therefore theoretically be used in clinical gene therapy protocols. Future challenges include the generation of lentiviral vectors that can express more than one transgene at high levels and the generation of safe permanent packaging cells for practical use in clinical gene therapy trials.
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