Musashi-2 sustains glucocorticoids resistance of infant KMT2A-rearranged Acute Lymphoblastic Leukemia via regulating the mitochondrial metabolism

MLL-rearranged B-cell Acute Lymphoblastic Leukemia occurring in infants (MLLr infant B-ALL) is a rare but very aggressive disease, associated with poor outcome. Infant patients with MLLr ALL are typically resistant to conventional chemotherapy and frequently relapse. The biological mechanisms involved in drug resistance and relapse occurrence of MLLr ALL are not completely understood, and the identification of novel therapeutic approaches to treat infant patients still remains an unmet clinical need. In this study we aimed at dissecting the functional role of the RNA-binding protein Musashi-2 (MSI2) in MLLr infant ALL, as a candidate biomarker of MLLr infant ALL and possibly involved in drug resistance, with a focus on potential therapeutic implications.