Targeted delivery of antisense-mediated exon skipping therapy in a DMD transgenic pig model
DMD 转基因猪模型中反义介导的外显子跳跃疗法的靶向递送
基本信息
- 批准号:331579
- 负责人:
- 金额:$ 1.27万
- 依托单位:
- 依托单位国家:加拿大
- 项目类别:Studentship Programs
- 财政年份:2014
- 资助国家:加拿大
- 起止时间:2014-12-01 至 2015-12-01
- 项目状态:已结题
- 来源:
- 关键词:
项目摘要
Duchenne muscular dystrophy (DMD), characterized by loss of muscle function, is one of the most common and deadly genetic disorders worldwide caused by mutations or alterations in the DMD gene. Currently, exon skipping therapy is one of the most promising
杜氏肌营养不良症 (DMD) 的特征是肌肉功能丧失,是世界范围内最常见和致命的遗传性疾病之一,由 DMD 基因突变或改变引起。目前,外显子跳跃疗法是最有前途的疗法之一
项目成果
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