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首页> 外文期刊>Molecular Therapy - Methods & Clinical Development >Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
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Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies

机译:产生基于VERO的包装细胞系,以产生用于临床基因治疗研究的SV40基因递送载体

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摘要

Replication-defective (RD) recombinant simian virus 40 (SV40)-based gene delivery vectors hold a great potential for clinical applications because of their presumed non-immunogenicity and capacity to induce immune tolerance to the transgene products in humans. However, the clinical use of {SV40} vectors has been hampered by the lack of a packaging cell line that produces replication-competent (RC) free {SV40} particles in the vector production process. To solve this problem, we have adapted the current {SV40} vector genome used for the production of vector particles and generated a novel Vero-based packaging cell line named SuperVero that exclusively expresses the {SV40} large T antigen. SuperVero cells produce similar numbers of {SV40} vector particles compared to the currently used packaging cell lines, albeit in the absence of contaminating {RC} {SV40} particles. Our unique {SV40} vector platform named {SVac} paves the way to clinically test a whole new generation of SV40-based therapeutics for a broad range of important diseases.
机译:复制缺陷(RD)重组猿猴病毒40(SV40)基因递送载体对临床应用具有很大的潜力,因为它们被推定的非免疫原性和诱导人类转基因产物的免疫耐受性。然而,{SV40}载体的临床用途被缺乏在载体生产过程中产生复制主管(RC)术术的包装态细胞系缺乏阻碍。为了解决这个问题,我们已经适应了用于生产载体粒子的电流{SV40}载体基因组,并产生一个名为SuperVero的新型vero的包装细胞系,其专门表达{sv40}大T抗原。与当前使用的包装细胞系相比,超比较细胞产生类似的{SV40}载体颗粒的数量,尽管在没有污染{rc} {sv40}粒子的情况下。我们名为{SVAC}的唯一{SV40}矢量平台铺平了临床测试全新一代基于SV40的治疗方法,以实现广泛的重要疾病。

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