Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination

• Main Authors: Lucile Hoch (Author), Nathalie Bourg (Author), Fanny Degrugillier (Author), Céline Bruge (Author), Manon Benabides (Author), Emilie Pellier (Author), Johana Tournois (Author), Gurvan Mahé (Author), Nicolas Maignan (Author), Jack Dawe (Author), Maxime Georges (Author), David Papazian (Author), Nik Subramanian (Author), Stéphanie Simon (Author), Pascale Fanen (Author), Cédric Delevoye (Author), Isabelle Richard (Author), Xavier Nissan (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Limb-girdle muscular dystrophy type R3 (LGMD R3) is a rare genetic disorder characterized by a progressive proximal muscle weakness and caused by mutations in the SGCA gene encoding alpha-sarcoglycan (α-SG). Here, we report the results of a mechanistic screening ascertaining the molecular mechanisms...