Alternative polyadenylation and splicing regulation in refractory juvenile dermatomyositis: CELF2 at the crossroads

Abstract

Juvenile dermatomyositis (JDM) is a rare, chronic autoimmune disease characterized by muscle weakness, skin rashes, and systemic inflammation. While corticosteroids and immunosuppressants remain the cornerstone of therapy, a significant subset of patients develop refractory disease, experiencing recurrent relapses and accumulating disability. For these patients, autologous hematopoietic stem cell transplantation (AHSCT) has emerged as a last-resort option, yet its molecular mechanisms of action remain poorly defined.