The Delaware Comprehensive Sickle Cell Research Center

特拉华州综合镰状细胞研究中心

• 批准号: 9056602
• 负责人: MARIE J. STUART
• 金额: $ 198.15万
• 依托单位: ALFRED I. DU PONT HOSP FOR CHILDREN
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-08-01 至 2019-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9056602
• 关键词: Adolescent; Affect; Area; Assessment tool; Basic Science; Biomedical Research; Centers of Research Excellence; Child; Childhood; Clinical; Clinical Data; Clinical Research; Core Facility; Data; Delaware; Electronics; Ensure; Equilibrium; Evaluation; Evidence based intervention; Family; Fatty Acids; Fish Oils; Foundations; Funding; Future; Genes; Genetic; Globin; Growth; Healthcare; In Vitro; Individual; Infant; Inflammation; Link; Oligonucleotides; Omega-3 Fatty Acids; Outcome; Pain; Patients; Pediatric Hospitals; Pediatric Research; Phase I/II Trial; Pilot Projects; Population; Protocols documentation; Psychosocial Assessment and Care; Public Health Informatics; Research; Research Infrastructure; Research Personnel; Resources; Risk; Sickle Cell; Sickle Cell Anemia; Stem cells; Stroke; Surrogate Markers; Work; cell growth; clinical care; data access; data integrity; genetic predictors; health disparity; high risk; meetings; nanofiber; next generation sequencing; novel; nuclease; programs; psychosocial; scaffold

The Delaware Comprehensive Sickle Cell Research Program (a consortium between A.I. duPont Hospital for Children and Delaware State Univ.) will build upon strengths in Biomedical Research, clinical care and recent recruitments to create a sustainable infrastructure supportive of target investigators and state-of-the-art research. Three Target Investigator proposals and two Pilots comprise a well balanced research portfolio spanning basic science, translational and clinical aspects of Sickle Cell Disease (SCD). Target Project I deals with gene editing of the Q> Globin gene using TAL Effector Nucleases and single stranded oligonucleotides. Target Project II includes an evaluation of surrogate biomarkers of SCD-related pain in children and a Phase I/II Trial of the n-3 Omega Fatty Acids in SCD-related pain and inflammation. Target Project III will use the Psychosocial Assessment Tool (PAT) to screen for risk in the pediatric population with SCD. The two Pilot Projects include studies on the in vitro growth of stem cells on nanofiber scaffolds, and the identification of genetic predictors of SCD-related stroke using next generation sequencing. The Overall Center Organization and Management Plan will incorporate key individuals, core facilities and resources previously funded through the IDeA Network, including the Center for Pediatric Research COBRE and the Delaware INBRE. A core will link advances in Health Informatics and electronic patient data recording with clinical research. Clinical data access and data integrity will provide a foundation for proposed and future clinical research. Strong Institutional support combined with federal dollars through the COBRE Program will ensure an outstanding program which will meet the ongoing needs of infants, children and adolescents with SCD and their families in Delaware. In addition, the research proposed will have a significant impact not only in the area of gene editing and in vitro stem cell growth but on SCD patient management in both the understanding and treatment of SCD related pain and stroke. The identification of the SCD affected family with high psychosocial risk will be also undertaken, facilitating appropriate evidence-based interventions that will diminish disparities in health care and outcomes.

特拉华州综合镰状细胞研究计划（A.I. 杜邦医院之间的联盟） 儿童和特拉华州立大学）将建立在生物医学研究、临床护理和 最近进行了招聘，以创建支持目标调查人员和最先进技术的可持续基础设施 研究。三个目标研究者提案和两个试点项目组成了一个均衡的研究组合 涵盖镰状细胞病（SCD）的基础科学、转化和临床方面。目标项目一 使用 TAL 效应核酸酶和单链处理 Q> 球蛋白基因的基因编辑 寡核苷酸。目标项目 II 包括评估 SCD 相关疼痛的替代生物标志物 儿童和 n-3 欧米茄脂肪酸治疗 SCD 相关疼痛和炎症的 I/II 期试验。目标 项目 III 将使用心理社会评估工具 (PAT) 来筛查患有以下疾病的儿科人群的风险 SCD。这两个试点项目包括纳米纤维支架上干细胞体外生长的研究，以及 使用下一代测序鉴定 SCD 相关中风的遗传预测因子。整体 中心组织和管理计划将包括关键人员、核心设施和资源 之前通过 IDeA 网络资助，包括儿科研究中心 COBRE 和 特拉华州 INBRE。核心将健康信息学和电子患者数据记录的进步与 临床研究。临床数据访问和数据完整性将为拟议和未来提供基础 临床研究。通过 COBRE 计划，强有力的机构支持与联邦资金相结合将 确保一项出色的计划能够满足婴儿、儿童和青少年的持续需求 SCD 和他们在特拉华州的家人。此外，拟议的研究将产生重大影响，而不是 仅在基因编辑和体外干细胞生长领域，但在 SCD 患者管理方面 了解和治疗 SCD 相关的疼痛和中风。 SCD受影响家庭的识别 还将采取具有高社会心理风险的措施，促进采取适当的循证干预措施， 将减少医疗保健和结果方面的差异。