Genetic engineering of T cells to express tumor antigen-specific T cell receptors (TCRs) is a promising approach in cancer immunotherapy. While the cytotoxic potential against cancer of these engineered TCR T cells is critical, their therapeutic efficacy also depends on their ability to migrate, infiltrate, proliferate, and persist within the complex tumor microenvironment (TME). This is...
Menin inhibitors represent a novel class of targeted therapies for acute myeloid leukemia (AML), particularly in patients with KMT2A rearrangements (KMT2A-r) and NPM1 mutations (NPM1mut). These inhibitors disrupt the Menin-KMT2A complex, silencing leukemogenic gene expression, promoting differentiation, and leading to disease eradication. However, the precise mechanisms by which the...
In vivo gene delivery has emerged as powerful tool for novel therapeutic concepts. A major challenge is the development of vectors capable of mediating highly selective gene transfer specifically into therapy-relevant cells. For this purpose, we have developed the concept of rational-based receptor-targeting of AAV vectors harnessing designed ankyrin repeat proteins (DARPins) in order to...
