Preclinical Gene Therapy in a Large Animal Model of DMD

DMD 大型动物模型的临床前基因治疗

• 批准号: 7509210
• 负责人: Xiao Xiao
• 金额: $ 13.84万
• 依托单位: UNIVERSITY OF PITTSBURGH AT PITTSBURGH
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-06-01 至 2009-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7509210
• 关键词: Animal Model; Animals; Biochemical; Canis familiaris; Cell membrane; Chronic; Clinical; Collaborations; Complementary DNA; Condition; Dependovirus; Duchenne muscular dystrophy; Dystrophin; Environment; Exhibits; Factor IX; Gene Delivery; Gene Expression; Gene Transfer; Genes; Genetic; Goals; Heart; Histopathology; Human; Immune; Immune response; Inflammation; Injection of therapeutic agent; Intramuscular; Intraperitoneal Injections; Invasive; Laboratories; LacZ Genes; Leg; Limb structure; Mediating; Methods; Missouri; Modeling; Mus; Muscle; Muscular Dystrophies; Myopathy; Non-Viral Vector; Normal Cell; Outcome; Pathology; Patients; Perfusion; Proteins; Purpose; Reporter; Reporter Genes; Route; Symptoms; Testing; Therapeutic Effect; Transducers; Universities; Viral; adeno-associated viral vector; gene therapy; immunogenic; improved; in vivo; male; mdx mouse; mini-dystrophin; novel; pre-clinical; response; size; vector