Sánchez-Hernández Sabina, Gutierrez-Guerrero Alejandra, Martín-Guerra Rocío, Cortijo-Gutierrez Marina, Tristán-Manzano María, Rodriguez-Perales Sandra, Sanchez Laura, Garcia-Perez Jose Luis, Chato-Astrain Jesus, Fernandez-Valades Ricardo, Carrillo-Galvez Ana Belén, Anderson Per, Montes Rosa, Real Pedro J, Martin Francisco, Benabdellah Karim
Genomic Medicine Department, GENYO, Centre for Genomics and Oncological Research, Pfizer-University of Granada-Andalusian Regional Government, PTS Granada, 18016 Granada, Spain.
Molecular Cytogenetics and Genome Editing Unit, Human Cancer Genetics Department, CNIO, Melchor Fernandez Almagro 3, 28029 Madrid, Spain.
Mol Ther Nucleic Acids. 2018 Dec 7;13:16-28. doi: 10.1016/j.omtn.2018.08.007. Epub 2018 Aug 18.
Integration-defective lentiviral vectors (IDLVs) have become an important alternative tool for gene therapy applications and basic research. Unfortunately, IDLVs show lower transgene expression as compared to their integrating counterparts. In this study, we aimed to improve the expression levels of IDLVs by inserting the IS2 element, which harbors SARs and HS4 sequences, into their LTRs (SE-IS2-IDLVs). Contrary to our expectations, the presence of the IS2 element did not abrogate epigenetic silencing by histone deacetylases. In addition, the IS2 element reduced episome levels in IDLV-transduced cells. Interestingly, despite these negative effects, SE-IS2-IDLVs outperformed SE-IDLVs in terms of percentage and expression levels of the transgene in several cell lines, including neurons, neuronal progenitor cells, and induced pluripotent stem cells. We estimated that the IS2 element enhances the transcriptional activity of IDLV LTR circles 6- to 7-fold. The final effect the IS2 element in IDLVs will greatly depend on the target cell and the balance between the negative versus the positive effects of the IS2 element in each cell type. The better performance of SE-IS2-IDLVs was not due to improved stability or differences in the proportions of 1-LTR versus 2-LTR circles but probably to a re-positioning of IS2-episomes into transcriptionally active regions.
整合缺陷型慢病毒载体(IDLVs)已成为基因治疗应用和基础研究的重要替代工具。不幸的是,与整合型慢病毒载体相比,IDLVs的转基因表达较低。在本研究中,我们旨在通过将含有SARs和HS4序列的IS2元件插入其长末端重复序列(LTRs)中来提高IDLVs的表达水平(SE-IS2-IDLVs)。与我们的预期相反,IS2元件的存在并未消除组蛋白去乙酰化酶引起的表观遗传沉默。此外,IS2元件降低了IDLV转导细胞中的附加体水平。有趣的是,尽管有这些负面影响,但在包括神经元、神经祖细胞和诱导多能干细胞在内的几种细胞系中,SE-IS2-IDLVs在转基因的百分比和表达水平方面优于SE-IDLVs。我们估计IS2元件可将IDLV LTR环的转录活性提高6至7倍。IS2元件在IDLVs中的最终效果将极大地取决于靶细胞以及IS2元件在每种细胞类型中的负面与正面效应之间的平衡。SE-IS2-IDLVs表现更好并非由于稳定性提高或1-LTR与2-LTR环比例的差异,而可能是由于IS2附加体重新定位到转录活性区域。