{"id":94,"date":"2021-06-14T12:25:35","date_gmt":"2021-06-14T10:25:35","guid":{"rendered":"https:\/\/semmelweis.hu\/hcemm-neurobiology\/?page_id=94"},"modified":"2026-02-06T14:37:43","modified_gmt":"2026-02-06T13:37:43","slug":"vector-core","status":"publish","type":"page","link":"https:\/\/semmelweis.hu\/hcemm-neurobiology\/core-facilities\/vector-core\/","title":{"rendered":"Vector Core"},"content":{"rendered":"

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Purpose<\/strong><\/p>\n

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The primary purpose of the Viral Vector Core is to use a Biosafety Level 2 laboratory at the Institute of Clinical Pathophysiology (EOK Laboratory 0.112), open to both Semmelweis University and academic working groups, for the production and preclinical use of viral vectors. In recent years, cell reprogramming technologies have revolutionised in vitro techniques and allowed to create and study many human-induced pluripotent stem cells (iPSCs) and directly reprogrammed disease-specific cellular subtypes (transdifferentiated, so-called induced cells). Notwithstanding, the Nobel Prize-winning CRISPR\/CAS9 gene editing technology was presented. Together, these combined methodological possibilities have opened up unprecedented research perspectives, mostly in genetic modelling, preclinical investigation and validation of complex human diseases. The new Viral Vector Core Facility provides a unique platform of state-of-the-art technologies for the Semmelweis University and all academic groups.<\/p>\n

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Background<\/strong><\/p>\n

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The usage for in vivo<\/em> validation of human\/mouse\/rat 2D or 3D primary cell cultures is a necessity for many clinical trials. Transfection of primary cells, specific tissues, organs is extremely challenging in the lab. Transduction, using viral vectors in in vitro<\/em> and in vivo<\/em> experimental models provides a widely used and well-controlled solution. Viral Vector Core Facilities are present at numerous universities to provide in-house viral vector production, which is undoubtedly a huge advantage in basic and applied research activities.<\/p>\n

The Viral Vector Core Facility at the Semmelweis University offers a shared organisational unit functioning as a platform. The platform provides researchers viral vectors in a unique way: from designing and cloning to production, and titration. The platform provides research teams with access to state-of-the-art vector technologies, preclinical and other translational research. These experiments will contribute to essential information that can lead to subsequent innovative clinical trials with high patenting potential.<\/p>\n

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Aims<\/strong><\/p>\n

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  1. Generation of lentiviral (overexpression of cDNA, shRNA, miRNA, etc.)<\/li>\n<\/ol>\n

    \u00a0 \u00a0 \u00a0 \u00a0 Design and cloning<\/p>\n

    \u00a0 \u00a0 \u00a0 \u00a0 Production, titration and quality control (QC) of viral vectors<\/p>\n

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    1. Generation of CRISPR\/CAS9 lentiviral vectors for gene modification, gene therapy<\/li>\n<\/ol>\n

      \u00a0 \u00a0 \u00a0 \u00a0 CRISPR KO, CRISPRi (CRISPR interference), CRISPRa (CRISPR activation)<\/p>\n

      \u00a0 \u00a0 \u00a0 \u00a0 Design and cloning<\/p>\n

      \u00a0 \u00a0 \u00a0 \u00a0 Production, titration and quality control (QC) of viral vectors<\/p>\n

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      Workplan<\/strong><\/p>\n

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      Researchers\/clinicians first deliver their plasmid of interest to the platform. It is also possible to design the vector plasmid via consultation. The platform will also help to select the right promoter, CRISPR technology and the most suitable vector design for in vivo<\/em> or in vitro<\/em> experiments. The Viral Vector Core Facility will start viral vector production which can take up 4 to 6 weeks (depending on cloning):<\/p>\n