New Gene Therapy Strategies for the Deletion of Exon 44 of Dystrophin Gene Based on Gene Editing by

• 作 者: Ping Li Yunzhi Pan Alice S. S.
• 机 构: CentreHospitalierdel’Université,LavalResearchCenter,DepartmentofMolecularDiagnosticsandBiopharma
• 出 处: 药物化学期刊(英文)
• 发表日期: 2013年1期
• 关键词: TALENs DMD GENE Therapy EXON SKIPPING TALENs DMD Gene Therapy Exon Skipping

Duchenne Muscular Dystrophy (DMD) is a severe childhood form of muscular dystrophy. Both the severe form and its milder form of Becker Muscular Dystrophy (BMD) are caused by the mutation of dystrophin gene. Different from some other genetic diseases such