Development of optimized adenovirus-based vectors for evaluation in a mouse model of Spinal Muscular Atrophy

开发基于腺病毒的优化载体，用于脊髓性肌萎缩症小鼠模型的评估

• 批准号: 279779
• 负责人: McFall Emily R
• 金额: $ 1.27万
• 依托单位: Ottawa Hospital Research Institute
• 依托单位国家: 加拿大
• 项目类别: Studentship Programs
• 财政年份: 2013
• 资助国家: 加拿大
• 起止时间: 2013-02-01 至 2014-02-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=279779&lang=en
• 关键词: Adenovirus; Dissection; Gene Therapy; In Vitro And In Vivo Infections; Ip And Iv Injections; Mouse Model; Solid And Liquid Overlays; Spinal Muscular Atrophy; Titers; Viral Purification

Spinal Muscular Atrophy (SMA) is a genetic disorder affecting 1 in 6000 live births. In its most severe form, children with SMA are never able to sit and succumb to the disease within the first 2 years of life. The disease is caused by mutations in the g

脊髓性肌萎缩症(SMA)是一种遗传性疾病，每6000名活产儿中就有一名受到影响。在最严重的情况下，患有SMA的儿童永远不能坐着并在生命的头两年内死于这种疾病。这种疾病是由G基因突变引起的。