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

• मुख्य लेखकों: Delphine Trochet (लेखक), Bernard Prudhon (लेखक), Lylia Mekzine (लेखक), Mégane Lemaitre (लेखक), Maud Beuvin (लेखक), Laura Julien (लेखक), Sofia Benkhelifa-Ziyyat (लेखक), Mai Thao Bui (लेखक), Norma Romero (लेखक), Marc Bitoun (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2022-03-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...