Diana Schenkwein

Designer gene transfer tools (vectors) are key in treating different inherited or acquired diseases safely with gene therapy. With the help of multilevel-optimized vectors based on the Human Immunodeficiency Virus 1 (HIV-1) and CRISPR/Cas, we pursue to develop an effective and safe gene therapy treatment to familial hypercholesterolemia and severe heart failure, both important diseases in Finland but also leading causes of deaths worldwide. Our research also includes the creation of safe and optimally efficient CAR T cells for cell therapy of cancer, studying the role of nucleoli in health and disease, characterizing genome-wide effects of transgene integration, and assessing the safety of gene transfer. We have shown that the natural tendency of lentivirus vectors (LVs) to integrate in a nearly random manner can be modified so that they are less likely to destroy important cellular genes or cause other unwanted side-effects. The modified vectors can also be harnessed for the delivery of desired proteins into target cells. Our approaches to improve the safety and efficiency of gene therapy treatments include multi-level targeting of LVs to the cells and tissues most relevant to achieve a curative outcome and through minimizing the risks for undesired side effects, including insertional mutagenesis and immunogenicity.

I am part of the The GeneCellNano consortium funded by the Academy of Finland’s Flagship Programme.