Improvement of CAR-T cell anti-leukemic effectorfunctions using CRISPR/Cas9 technology andCliniMACS Prodigy system

01.01.2021 bis 31.12.2022

Leukemia is one of the harshest diseases of our time. Because of its worldwide prevalence and heterogeneity, many therapeutic approaches are currently being developed. One of the most promising strategies involves CAR-T cells, living drugs able to efficiently target and destroy cancer cells. The aim of this doctoral project consists on the set-up of a biomedical platform to generate highly maturated universal CAR-T cells in GMP quality for rapid clinical translation. To proceed, we will take advantage of the well-known CRISPR/Cas9 system to generate and optimize suitable CAR-T cell expansion protocols. By targeting and disrupting inhibitory immune checkpoints, we expect to enhance the anti-leukemic function of the treatment. Safety and efficiency of genetic engineering will be critically tested with innovative techniques such as GUIDEseq to offer a solid, low-risk alternative to the traditional anticancer therapies. The work will be established on the automated CliniMACS Prodigy® system, a state-of-the-art device that will ensure the excellence of the potential treatment. We hypothesize that the success of the proposed project could imply a new benchmark in ranslational research and would simplify the development of personalized mmunotherapy.