Benefits of therapy by dynamin-2-mutant-specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy

• Main Authors: Delphine Trochet (Author), Bernard Prudhon (Author), Lylia Mekzine (Author), Mégane Lemaitre (Author), Maud Beuvin (Author), Laura Julien (Author), Sofia Benkhelifa-Ziyyat (Author), Mai Thao Bui (Author), Norma Romero (Author), Marc Bitoun (Author)
• Format: Book
• Published: Elsevier, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Dominant dynamin 2 (DNM2) mutations are responsible for the autosomal dominant centronuclear myopathy (AD-CNM), a rare progressive neuromuscular disorder ranging from severe neonatal to mild adult forms. We previously demonstrated that mutant-specific RNA interference is an efficient therapeutic str...