Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations

• Main Authors: Swati Dudhal (Author), Lylia Mekzine (Author), Bernard Prudhon (Author), Karishma Soocheta (Author), Bruno Cadot (Author), Kamel Mamchaoui (Author), Delphine Trochet (Author), Marc Bitoun (Author)
• Format: Book
• Published: Elsevier, 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Dominant centronuclear myopathy (CNM) is a rare form of congenital myopathy associated with a wide clinical spectrum, from severe neonatal to milder adult forms. There is no available treatment for this disease due to heterozygous mutations in the DNM2 gene encoding Dynamin 2 (DNM2). Dominant DNM2 m...