The Delaware Comprehensive Sickle Cell Research Center

特拉华州综合镰状细胞研究中心

• 批准号: 9274317
• 负责人: MARIE J. STUART
• 金额: $ 192.92万
• 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/9274317
• 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; Ensure; 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; Pilot Projects; Population; Protocols documentation; Psychosocial Assessment and Care; Public Health Informatics; Recruitment Activity; 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; nanofiber; next generation sequencing; novel; nuclease; programs; psychosocial; public health relevance; scaffold

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州立大学）将建立在生物医学研究，临床护理和最近的招聘优势，以创建一个可持续的基础设施，支持目标研究者和最先进的研究。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，涵盖了镰状细胞病（SCD）的基础科学、转化和临床方面。目标项目I涉及使用TAL效应核酸酶和单链寡核苷酸对Q>珠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和n-3 ω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。这两项先导计划包括研究干细胞在人造血管支架上的体外生长，以及利用下一代测序技术鉴定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INBRE。一个核心将把健康信息学和电子病人数据记录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，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，这将满足特拉华州患有SCD的婴儿、儿童和青少年及其家庭的持续需求。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领域，但在理解和治疗SCD相关疼痛和中风方面的SCD患者管理。还将识别患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，以缩小医疗保健和结果的差异。