GENE THERAPY FOR SICKLE CELL DISEASE

镰状细胞病的基因治疗

• 批准号: 8150164
• 负责人: Punam Malik
• 金额: $ 20.93万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-04-01 至 2012-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8150164
• 关键词: Accounting; Affect; Allogenic; Anemia; Autologous; Azacitidine; Bone Marrow; Butyrates; CD34 gene; Cells; Chromatin; Clinic; Clinical; Clinical Research; Codon Nucleotides; Collection; Decitabine; Elements; Engraftment; Equilibrium; Erythroid; Erythroid Cells; Erythropoiesis; Fanconi' s Anemia; Fetal Hemoglobin; Gene Transfer; Genes; Genetic; Globin; Goals; Harvest; Hematopoietic stem cells; Hemoglobin F Disease; Human; In Vitro; Laboratories; Lentivirus Vector; Life; Malignant Neoplasms; Mediating; Modeling; Mus; Patients; Peripheral Blood Stem Cell; Pharmaceutical Preparations; Phase; Phenotype; Production; Protocols documentation; RNA Interference; Recombinants; Reporting; Safety; Sickle Cell Anemia; Sickle Hemoglobin; Source; Stem cell transplant; Stem cells; Thalassemia; Therapeutic; Time; Toxic effect; beta Globin; beta Thalassemia; chemotherapy; conditioning; design; gene therapy; gene therapy clinical study; genetic manipulation; hybrid gene; hydroxyurea; improved; in vivo Model; pilot trial; polymerization; pre-clinical; prevent; safety study; sickling; small hairpin RNA; vector