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首页> 外文期刊>Biotechnology Progress >Large-scale processing of recombinant retroviruses for gene therapy[Review]
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Large-scale processing of recombinant retroviruses for gene therapy[Review]

机译：用于基因治疗的重组逆转录病毒的大规模加工[综述]

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摘要

Gene therapy is a new therapeutic modality with the potential of treating inherited and acquired diseases. Several viral and physicochemical vehicles have been used for the transfer of genes to mammalian cells, but recombinant retroviruses are used in the majority of gene therapy clinical trials today. In this communication, we review the major concerns associated with the large-scale production and processing of retroviral particles. While some of the current processes for manufacturing recombinant proteins will be applicable to recombinant retroviruses, the instability, sensitivity to inhibitors, complexity, and size of retroviral particles require that new technologies be designed and evaluated. Here, we examine those issues critical to the design of strategies for production, concentration, and purification as well as formulation and storage of recombinant retroviruses. Processes for large-scale manufacturing of recombinant retroviruses that can produce high gene transfer efficiencies will have significant impact on the clinical implementation of gene therapy.
机译：基因治疗是一种新的治疗方法，具有治疗遗传性和后天性疾病的潜力。几种病毒和物理化学媒介物已被用于将基因转移至哺乳动物细胞，但是当今大多数基因疗法临床试验中都使用了重组逆转录病毒。在本交流中，我们回顾了与大规模生产和加工逆转录病毒颗粒有关的主要问题。尽管目前一些生产重组蛋白的方法将适用于重组逆转录病毒，但逆转录病毒颗粒的不稳定性，对抑制剂的敏感性，复杂性和大小要求设计和评估新技术。在这里，我们研究了那些对于生产，浓缩和纯化以及重组逆转录病毒的配制和储存策略设计至关重要的问题。大规模生产可产生高基因转移效率的重组逆转录病毒的过程将对基因治疗的临床实施产生重大影响。

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