Toscano Miguel G, van der Velden Jeroen, van der Werf Sybrand, Odijk Machteld, Roque Ana, Camacho-Garcia Rafael J, Herrera-Gomez Irene G, Mancini Irene, de Haan Peter
Amarna Therapeutics SL, Cartuja Technology Park, Leonardo da Vinci Avenue 18, 41092 Sevilla, Spain.
Amarna Therapeutics B.V., J.H. Oortweg 21, 2333 CH Leiden, the Netherlands.
Mol Ther Methods Clin Dev. 2017 Jul 5;6:124-134. doi: 10.1016/j.omtm.2017.06.007. eCollection 2017 Sep 15.
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 SV paves the way to clinically test a whole new generation of SV40-based therapeutics for a broad range of important diseases.
复制缺陷型（RD）重组猿猴病毒40（SV40）基因递送载体因其假定的非免疫原性以及在人类中诱导对转基因产物免疫耐受的能力，在临床应用方面具有巨大潜力。然而，SV40载体的临床应用因在载体生产过程中缺乏能产生无复制能力（RC）的SV40颗粒的包装细胞系而受到阻碍。为解决这一问题，我们对当前用于生产载体颗粒的SV40载体基因组进行了改造，并构建了一种新型的基于非洲绿猴肾细胞（Vero）的包装细胞系，名为SuperVero，它专门表达SV40大T抗原。SuperVero细胞产生的SV40载体颗粒数量与目前使用的包装细胞系相似，尽管不存在污染性的RC SV40颗粒。我们独特的名为SV的SV40载体平台为临床测试新一代基于SV40的针对多种重要疾病的治疗方法铺平了道路。
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