Fernández-Frías Iván, Pérez-Luz Sara, Díaz-Nido Javier
Departamento Biología Molecular and Centro de Biología Molecular "Severo Ochoa" (UAM-CSIC), Universidad Autónoma de Madrid, 28049 Madrid.
Instituto Investigación Sanitaria Puerta de Hierro-Majadahonda, Madrid, Spain.
Mol Ther Methods Clin Dev. 2020 Mar 13;17:491-496. doi: 10.1016/j.omtm.2020.03.005. eCollection 2020 Jun 12.
Herpes simplex virus 1 (HSV-1)-derived amplicon vectors are unique in their ability to accommodate large DNA molecules allowing whole genomic loci to be included with all of their regulatory elements. Additional advantages of these amplicons include their minimal toxicity and ability to persist as episomes, with negligible risk of insertional mutagenesis, being particularly well-suited for gene therapy of neurological disorders due to their outstanding ability to deliver genes into neurons and other neural cells. However, extensive gene therapy application has been hindered by difficulties in vector production. This work improved HSV-1 amplicons production by genetic modification of the packaging cell line and optimization of the culture medium. A stably-transfected Vero 2-2 cell line overexpressing the anti-apoptotic Bcl-2 protein was generated, exhibiting an increased resistance to apoptosis, prolonged culture duration, and a significant improvement in viral vector production. Additionally, supplementation of the growth medium with antioxidants, polyamines, amino acids, and reduced glutathione further increased the yield of packaged amplicon vectors. With these modifications, HSV-1 amplicons could be isolated from culture supernatants instead of cell lysates, leading to vector preparations with higher titer and purity and paving the way for generation of stable cell lines that are capable of continuous herpesviral vector production.
单纯疱疹病毒1型(HSV-1)衍生的扩增子载体在容纳大DNA分子的能力方面独具特色,能够包含整个基因组位点及其所有调控元件。这些扩增子的其他优势包括毒性极小,能以附加体形式持续存在,插入诱变风险可忽略不计,由于其向神经元和其他神经细胞传递基因的卓越能力,特别适合用于神经系统疾病的基因治疗。然而,载体生产方面的困难阻碍了其在基因治疗中的广泛应用。这项工作通过对包装细胞系进行基因改造和优化培养基来改进HSV-1扩增子的生产。构建了稳定转染的过表达抗凋亡Bcl-2蛋白的Vero 2-2细胞系,该细胞系对凋亡的抗性增强,培养持续时间延长,病毒载体产量显著提高。此外,在生长培养基中添加抗氧化剂、多胺、氨基酸和还原型谷胱甘肽进一步提高了包装扩增子载体的产量。通过这些改进,可从培养上清液而非细胞裂解物中分离HSV-1扩增子,从而获得更高滴度和纯度的载体制剂,为能够持续生产疱疹病毒载体的稳定细胞系的产生铺平了道路。