A Novel, Non-Cytotoxic, Epigenetic Therapeutic for Sickle Cell Disease

镰状细胞病的新型非细胞毒性表观遗传疗法

• 批准号: 9755493
• 负责人: JOSEPH DESIMONE
• 金额: $ 75万
• 依托单位: DENOVOTHERAPIES, LLC
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-04-24 至 2020-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9755493
• 关键词: Address; Adoption; Adverse event; Biological Availability; Chemicals; Chemistry; Clinic; Clinical; Clinical Research; Clinical Trials; Coagulation Process; Consumption; Cytidine Deaminase; Cytidine Deaminase Inhibitor; Cytostatics; DNA Modification Methylases; Data; Decitabine; Disease; Dose; Drug Combinations; Dysmyelopoietic Syndromes; Effectiveness; Enzymes; Epigenetic Process; Erythrocytes; FDA approved; Fetal Hemoglobin; Functional disorder; Generic Drugs; Genes; Goals; Hemoglobin; Hemolysis; Hour; Hydrogenation; In Vitro; Interruption; Intestines; Ion-Exchange Chromatography Procedure; Laboratory Markers; Life; Liver; Modeling; Modification; Morbidity - disease rate; Movement; Mus; Mutate; National Heart, Lung, and Blood Institute; National Institute of Diabetes and Digestive and Kidney Diseases; Oral; Papio; Pathway interactions; Patients; Pharmaceutical Preparations; Pharmacologic Substance; Pharmacology; Pharmacy facility; Phase; Phase I Clinical Trials; Placebos; Plasma; Polymers; Positioning Attribute; Powder dose form; Prevalence; Process; Proteins; Rare Diseases; Reaction; Refractory; Reporting; Rhodium; Risk; S Phase; Safety; Scheme; Sickle Cell Anemia; Sickle Hemoglobin; Small Business Innovation Research Grant; Small Intestines; Stomach; Tablets; Tetrahydrouridine; Therapeutic; Time; United States National Institutes of Health; Uridine; catalyst; commercialization; cost; cytotoxic; cytotoxicity; drug development; gamma Globin; hydroxyurea; in utero; in vivo; mortality; novel; process optimization; reconstitution