Gamma Globin Gene Therapy Using In Vivo Selection

使用体内选择的伽马珠蛋白基因治疗

• 批准号: 7538839
• 负责人: DEREK A PERSONS
• 金额: $ 42.35万
• 依托单位: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-04-01 至 2008-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7538839
• 关键词: Acute Erythroblastic Leukemia; Area; Biological Assay; Bone Marrow; Bone Marrow Transplantation; Cells; Chimera organism; Chimerism; Clinical; Clinical Data; Clinical Trials; Data; Development; Disease model; Drug resistance; Enhancers; Erythrocytes; Erythroid Cells; Evaluation; Functional disorder; Future; Gene Transfer; Generations; Genes; Globin; Goals; Hematopoietic stem cells; Human; In Vitro; Lentivirus Vector; Literature; Locus Control Region; Longevity; MEL Gene; Marines; Methyltransferase; Minor; Modeling; Modification; Mus; Normal Cell; Numbers; Organ; Patients; Performance; Pharmaceutical Preparations; Population; Positioning Attribute; Proteins; Rab8 protein; Relative (related person); Screening procedure; Series; Sickle Cell; Sickle Cell Anemia; Stem cells; System; Testing; Therapeutic; Therapeutic Effect; Therapy Clinical Trials; Time; Transgenes; Treatment Efficacy; Variant; base; beta Thalassemia; conditioning; design; gamma Globin; gene therapy; improved; in vivo; mouse model; pressure; research study; sickling; stem; success; vector