The Delaware Comprehensive Sickle Cell Research Center

特拉华州综合镰刀细胞研究计划(A.I.杜邦医院之间的联盟 为儿童和特拉华州立大学。)将在生物医学研究、临床护理和 最近的招聘，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。三个目标调查员提案和两个飞行员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、翻译和临床方面。目标项目I 利用TAL效应核酸酶和单链DNA对Q&gt；珠蛋白基因进行基因编辑 寡核苷酸。目标项目II包括对SCD相关疼痛的替代生物标记物的评估 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)来筛查儿童人群中的风险 SCD。这两个试点项目包括干细胞在纳米纤维支架上的体外生长研究，以及 应用下一代测序技术确定SCD相关卒中的遗传预测因子。整体而言 中心组织和管理计划将纳入关键个人、核心设施和资源 以前由IDEA网络资助，包括儿科研究中心Cobre和 特拉华州因布雷。一个核心将把健康信息学和电子患者数据记录方面的进展与 临床研究。临床数据访问和数据完整性将为计划和未来提供基础 临床研究。通过科布雷计划，强有力的机构支持与联邦资金相结合，将 确保一个出色的计划，满足婴幼儿、儿童和青少年的持续需求 特拉华州的SCD和他们的家人。此外，拟议的研究将产生重大影响，不 仅在基因编辑和体外干细胞生长方面，但在SCD患者管理方面 对SCD相关疼痛和中风的认识和治疗。SCD患者家系的鉴定 还将开展具有较高心理社会风险的干预，促进适当的循证干预 将缩小医疗保健和结果方面的差距。

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.