PHARMACOLOGIC/GENE THERAPY APPROACHES TO TREATMENT OF HEMOGLOBIN DISORDERS

治疗血红蛋白疾病的药理学/基因治疗方法

• 批准号: 3741020
• 负责人: GEORGE ATWEH
• 金额: --
• 依托单位: MOUNT SINAI SCHOOL OF MEDICINE OF CUNY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3741020
• 关键词: arginine; blood disorder chemotherapy; gene therapy; hemoglobin F; human subject; human therapy evaluation; intravenous administration; sickle cell anemia

The purpose of this pilot study is to determine the range of responses to intravenous arginine butyrate as gauged by the increase in fetal hemoglobin production in patients with sickle cell anemia and beta-thalassemia syndromes.

这项试点研究的目的是确定反应的范围 静脉注射精氨酸丁酸盐，以胎儿 镰状细胞性贫血患者的血红蛋白生成， β地中海贫血综合征。