The Delaware Comprehensive Sickle Cell Research Center

描述(由申请者提供)：特拉华州综合镰状细胞研究项目(由杜邦儿童医院和特拉华州立大学组成的联合体)将在生物医学研究、临床护理和最近的招聘方面建立优势，以创建支持目标调查人员和最先进研究的可持续基础设施。三个目标调查者提案和两个试点组成了一个平衡良好的研究组合，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涉及使用TAL效应核酸酶和单链寡核苷酸对Q&gt；珠蛋白基因进行基因编辑。目标项目II包括评估儿童SCD相关疼痛的替代生物标记物，以及n-3欧米茄脂肪酸治疗SCD相关疼痛和炎症的L/11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SCD的儿科人群中的风险。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，以及使用下一代测序识别与SCD相关的中风的遗传预测因素。整个中心组织和管理计划将纳入以前通过IDEA网络资助的关键个人、核心设施和资源，包括儿科研究中心和特拉华州INBRE。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联系起来。临床数据访问和数据完整性将为拟议和未来的临床研究提供基础。通过Cobre计划提供的强有力的机构支持和联邦资金将确保一个出色的计划，满足特拉华州患有SCD的婴儿、儿童和青少年及其家人的持续需求。此外，拟议的研究不仅将在以下方面产生重大影响 在基因编辑和干细胞体外生长领域，但在SCD患者管理方面，无论是对SCD相关疼痛和中风的理解和治疗都是如此。还将对有高心理社会风险的受SCD影响的家庭进行识别，促进以证据为基础的适当干预措施，以缩小卫生保健和结果的差异。

DESCRIPTION (provided by applicant): 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 l/ll 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.