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

• 主要な著者: Swati Dudhal (著者), Lylia Mekzine (著者), Bernard Prudhon (著者), Karishma Soocheta (著者), Bruno Cadot (著者), Kamel Mamchaoui (著者), Delphine Trochet (著者), Marc Bitoun (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2022-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: 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...