Expression of introduced genetic sequences in hematopoietic cells following retroviral-mediated gene transfer.
Expression of introduced genetic sequences in hematopoietic cells following retroviral-mediated gene transfer.
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逆转录病毒介导的基因转移后引入的基因序列在造血细胞中的表达。
作者：
David A. Williams
使用逆转录病毒载体可以有效地将基因转移到各种哺乳动物细胞中。过去6年的研究重点是利用逆转录病毒载体将基因转移到造血细胞中。这些转导的细胞可能会用于影响血细胞的严重遗传疾病的基因治疗。尽管早期对多种基因序列在造血细胞中的转移和表达持乐观态度，但在实现引入基因在造血干细胞后代中稳定和长期表达的目标方面，进展缓慢，令人沮丧，而且只取得了部分成功。这种缓慢的进展部分是由于缺乏对原代细胞中基因调控控制的理解，同时也是由于小鼠和大型动物中造血干细胞生物学的复杂性。本文综述了将基因导入造血细胞的研究进展，以及在将基因移植方法应用于人类骨髓源性细胞疾病之前仍存在的困难。
The use of retroviral vectors allows efficient transfer of genes into a variety of mammalian cells. A focus of research over the past 6 years has been the use of retroviral vectors to effect gene transfer into hematopoietic cells. These transduced cells might then be used for gene therapy of severe genetic diseases affecting blood cells. In spite of early optimism concerning the transfer and expression of a variety of gene sequences in hematopoietic cells, progress in obtaining the goal of stable and long-term expression of introduced genes in progeny of hematopoietic stem cells has been slow, frustrating, and only partially successful. This slow progress has been due, in part, to lack of understanding of the control of gene regulation in primary cells but also to the complexity of hematopoietic stem cell biology in both murine and large animal species. This review attempts to summarize the progress that has been made in the expression of genes introduced into hematopoietic cells and the difficulties still remaining before meaningful application of gene transfer methods can be expected to cure human diseases of bone marrow-derived cells.
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逆转录病毒载体的增强子序列决定多能造血祖细胞和定向红系细胞中基因的表达。
DOI：
10.1073/pnas.84.23.8662
发表时间：
1987
影响因子：
11.1
作者：
Holland,CA;Anklesaria,P;Sakakeeny,MA;Greenberger,JS
通讯作者：
Greenberger,JS
基因转移至人造血细胞系后，人腺苷脱氨酶从各种强启动子的表达。
影响因子：
20.3
作者：
Hock,RA;Miller,AD;Osborne,WR
通讯作者：
Osborne,WR
限制整合重组逆转录病毒在原代而非永生化小鼠造血干细胞中的表达。
影响因子：
20.3
作者：
Williams,DA;Lim,B;Spooncer,E;Longtine,J;Dexter,TM
通讯作者：
Dexter,TM
移植逆转录病毒感染的造血干细胞的小鼠体内人腺苷脱氨酶的长期表达。
DOI：
10.1073/pnas.86.22.8892
发表时间：
1989
影响因子：
11.1
作者：
Lim,B;Apperley,JF;Orkin,SH;Williams,DA
通讯作者：
Williams,DA
逆转录病毒载体介导的基因转移到人类造血祖细胞中。
DOI：
10.1126/science.3864246
发表时间：
1985
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Gruber,HE;Finley,KD;Hershberg,RM;Katzman,SS;Laikind,PK;Seegmiller,JE;Friedmann,T;Yee,JK;Jolly,DJ
通讯作者：
Jolly,DJ