A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy

• Autors principals: Virginie Mariot (Autor), Romain Joubert (Autor), Anne-Charlotte Marsollier (Autor), Christophe Hourdé (Autor), Thomas Voit (Autor), Julie Dumonceaux (Autor)
• Format: Llibre
• Publicat: Elsevier, 2020-12-01T00:00:00Z.
• Matèries: article
• Accés en línia: Connect to this object online.

Facioscapulohumeral dystrophy (FSHD) is characterized by a loss of repressive epigenetic marks leading to the aberrant expression of the DUX4 transcription factor. In muscle, DUX4 acts as a poison protein though the induction of multiple downstream genes. So far, there is no therapeutic solution for...