ZPR1 regulation of SMN expression in spinal muscular atrophy


  • The zinc finger protein ZPR1 binds to RNA polymerase II and upregulates SMN2 expression in a mouse model of spinal muscular atrophy (SMA) and in patient-derived cells.

Why this matters

    Low SMN protein levels lead to DNA damage and neurodegeneration in SMA, better understanding the transcriptional role of ZPR1 to increase SMN2 production of SMN protein provides a new therapeutic target for treating SMA.