The mission of the Vector and Genome Engineering Facility (VGEF), an innovative academic scientific core facility of the Children’s Medical Research Institute, is to support basic and translational research in vector-based gene therapy and genome engineering. In addition to being a service facility that provides investigators the access to the latest viral-vector and genome engineering technologies, VGEF is also active in developing new, and improving existing, technologies that will empower its customers and the wider scientific community.
The VGEF facility offers customized preparations of lentiviral (LV), adenoviral (Ad), and adeno-associated viral (AAV) vectors, at purities and production scales tailored to the needs of individual projects to facilitate both in vitro and in vivo experimentation, as well as a range of ready-to-use AAV and LV stocks.
The VGEF also offers wide range of genome-editing services in immortalized lines and stem cells of human and animal origin. VGEF closely follows and utilizes the latest advances in genome editing technologies. Currently, VGEF uses a multitude of plasmid- and vector-based CRISPR/Cas systems based on active or inactive nucleases or nickase for precise genome or epigenetic modifications. We are also expending our services into RNA editing, endonuclease free editing using AAV-driven homology directed repair (HDR), as well as into editing with the use of Cas9 protein and Cas9 mRNA expression methods.
In alignment with our goal to promote vector-based and genome engineering technologies, we offer consultation in study design and technical support in the selection of appropriate tools for specific experimental needs, as well as hands-on training opportunities. Beyond our academic and commercial services, we also engage in collaborative studies that are aligned with our research focus in AAV biology, vector development and genome engineering.