Work

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

Year: 2025

Type: article

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.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102725

Authors: Riccardo Varrica, Laura Lentini

Institution: University of Palermo

Language: English

Topic: Inflammatory Myopathies and Dermatomyositis

Subfield: Epidemiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900