The Delaware Comprehensive Sickle Cell Research Center

特拉华州综合镰状细胞研究计划（A.I.杜邦医院 儿童和特拉华州州立大学）将建立在生物医学研究，临床护理和 最近的招聘，以建立一个可持续的基础设施，支持目标调查人员和最先进的 research.三个目标研究者提案和两个试点项目构成了一个平衡的研究组合 涵盖镰状细胞病（SCD）的基础科学、转化和临床方面。目标项目I 涉及使用TAL效应核酸酶和单链DNA酶对Q>珠蛋白基因进行基因编辑。 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I/II期试验的n-3欧米茄脂肪酸在SCD相关的疼痛和炎症。目标 项目III将使用心理社会评估工具（PAT）筛查儿科人群的风险， SCD。这两项试验计划包括研究干细胞在人造血管支架上的体外生长，以及 使用下一代测序鉴定SCD相关卒中的遗传预测因子。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 以前通过IDEA网络资助，包括儿科研究中心COBRE和 特拉华州INBRE。一个核心将把健康信息学和电子病人数据记录的进步与 临床研究临床数据访问和数据完整性将为拟议和未来的 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， 确保一个出色的方案，将满足婴儿，儿童和青少年的持续需求， SCD和他们的家人在特拉华州。此外，拟议的研究将产生重大影响， 仅在基因编辑和体外干细胞生长领域， 了解和治疗SCD相关疼痛和中风。SCD受影响家庭的识别 还将对具有高心理社会风险的人进行干预，促进适当的循证干预措施， 将缩小医疗保健和结果的差距。

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.