The Delaware Comprehensive Sickle Cell Research Center

特拉华州综合镰状细胞研究中心

• 批准号: 8898133
• 负责人: MARIE J. STUART
• 金额: $ 201.5万
• 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/8898133
• 关键词: Delaware; Comprehensive; Sickle; Cell; Research

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.

描述（由申请人提供）：特拉华州综合镰状细胞研究计划（A.I.杜邦儿童医院和特拉华州立大学之间的联盟）将利用生物医学研究、临床护理和最近招募的优势，创建支持目标的可持续基础设施调查人员和最先进的研究。三项目标研究者提案和两项试点项目构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 I 使用 TAL 效应核酸酶和单链寡核苷酸对 Q> 球蛋白基因进行基因编辑。目标项目 II 包括对儿童 SCD 相关疼痛的替代生物标志物的评估以及 n-3 欧米茄脂肪酸在 SCD 相关疼痛和炎症中的 I/II 期试验。目标项目 III 将使用心理社会评估工具 (PAT) 筛查患有 SCD 的儿科人群的风险。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。总体中心组织和管理计划将纳入先前通过 IDeA 网络资助的关键人员、核心设施和资源，包括儿科研究中心 COBRE 和特拉华州 IBRE。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联系起来。临床数据访问和数据完整性将为拟议的和未来的临床研究奠定基础。通过 COBRE 计划，强有力的机构支持与联邦资金相结合，将确保该计划出色，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，拟议的研究不仅会产生重大影响 基因编辑和体外干细胞生长领域，但在 SCD 患者管理方面，包括了解和治疗 SCD 相关疼痛和中风。还将确定具有高心理社会风险的 SCD 受影响家庭，促进适当的循证干预措施，从而减少医疗保健和结果方面的差异。