Versatile retroviral vectors for potential use in gene therapy.
Versatile retroviral vectors for potential use in gene therapy.
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DOI：
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发表时间：
1994-03
期刊：
影响因子：
5.1
通讯作者：
R. Hawley;F. Lieu;A. Fong;T. Hawley
中科院分区：
文献类型：
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作者：
R. Hawley;F. Lieu;A. Fong;T. Hawley
描述了一组逆转录病毒载体，其将功能基因高效转导到未分化的鼠胚胎和造血细胞中的能力使其非常适合于用鼠模型进行临床前研究。多个独特的克隆位点允许将基因插入载体中，使得不存在选择标记，或者包括新霉素磷酸转移酶（neo）基因、潮霉素B磷酸转移酶（hph）基因或嘌呤霉素N-乙酰转移酶（pac）基因作为显性作用的选择标记。由于病毒gag区中显示改善病毒RNA的腺苷酸化的序列已被修饰以防止病毒蛋白质合成，并且所有env序列已被去除以通过与包装DNA的同源重组消除辅助病毒的产生，因此这些载体可能证明在人类基因治疗方案中有用。
A set of retroviral vectors is described whose capacity for high efficiency transduction of functional genes into undifferentiated murine embryonic and haematopoietic cells makes them ideally suited for preclinical studies with murine models. Multiple unique cloning sites permit insertion of genes into the vectors such that no selectable marker exists or either the neomycin phosphotransferase (neo) gene, the hygromycin B phosphotransferase (hph) gene or the puromycin N-acetyl transferase (pac) gene is included as a dominantly acting selectable marker. Because the sequences in the viral gag region shown to improve the encapsidation of viral RNA have been modified to prevent viral protein synthesis and all env sequences have been removed to eliminate helper virus production by homologous recombination with packaging DNA, these vectors might prove useful in human gene therapy protocols.