Targeting CD74 for familial platelet disorder

Inhibiting the cell surface MIF receptor CD74, or its downstream JAK-1, JAK-2 and mTOR signaling, could help treat RUNX1 mutation-driven familial platelet disorder, which is a risk factor for AML, by increasing bone marrow platelet production without altering hematopoietic stem and progenitor cell (HSPC) numbers.

Single-cell RNA sequencing comparing bone marrow samples from familial platelet disorder patients and individuals without the disorder identified higher CD74 expression in HSPCs from patients. ...