Boston Comprehensive Sickle Cell Center

波士顿综合镰状细胞中心

• 批准号: 7343539
• 负责人: Martin H. Steinberg
• 金额: $ 55.11万
• 依托单位: BOSTON MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-07-01 至 2012-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7343539
• 关键词: Boston; Comprehensive; Sickle; Cell; Center

DESCRIPTION (provided by applicant): The Boston Comprehensive Sickle Cell Center (BCSCC) saw major reorganization over the past six years. In this competitive renewal application, while core center leadership remains unchanged, we have elected to build on the strengths of investigators available to BCSCC by making major changes in direction. We maintain the belief that individual investigator projects remain a critical part of the BCSCC mission and therefore have brought new investigators into the BCSCC, refocusing our bench and clinical research activities to include patient services research, translational studies of fetal hemoglobin (HbF) induction, further our understanding the genetic basis of red cell density regulation and examine the role of glucose-6-phosphate dehydrogenase (G6PD) and the modulation of endothelial activity. We are able to do this under current budget restaints because all of our project investigator have independent R01 or equivalent support that provides an important infrastructure upon which to build center activities. We will also build on our past successes in patient recruitment for collaborative clinical trials and we propose a clinical trial aimed at the prevention of hypovitaminosis D and its resulting bone disease. Specific Aims: 1. Carry out novel therapeutic trials in sickle cell anemia and HbSC disease by assembling a large Clinical Core of patients as candidates for Phase I and II clinical trials 2. Conduct innovative studies of: the genetic determinants of sickle erythrocyte hydration; endothelial function and G6PD deficiency; HbF induction by a novel oral agent; improving the delivery of comprehensive preventive services for children with sickle cell disease; and, improving bone health with vitamin D and calcium treatment 3. Provide high quality clinical care for all BCSCC patients with education and counseling for patients, education and research training for medical professionals and genetic testing for hemoglobinopathies 4. Meld the components of the BCSCC into a cohesive system to increase its effectiveness beyond the sum of its individual projects, encouraging interactions with other regional and national sickle cell centers.

描述（由申请人提供）：波士顿综合镰状细胞中心（BCSCC）在过去六年中进行了重大重组。在此竞争性更新的应用程序中，尽管核心中心领导层保持不变，但我们选择在BCSCC可用的调查人员的优势基础上，通过重大改变方向。我们坚持认为，个别研究者的项目仍然是BCSCC任务的关键部分，因此将新的研究人员带入BCSCC，重新调整了我们的长凳和临床研究活动，以包括患者服务研究，胎儿血红蛋白的翻译研究（HBF）诱导，进一步了解了红细胞密度调节的遗传和glucose demlose-deplose（G6-磷酸盐）的作用（Glucose-deplode）（Glucose-6-磷酸盐）的作用（G6-磷酸化的作用）（ 活动。我们能够根据当前的预算修订来执行此操作，因为我们所有的项目调查员都有独立的R01或同等支持，提供了重要的基础设施来建立中心活动。我们还将以我们过去在患者招募进行协作临床试验中取得的成功，并提出一项旨在预防降低症D及其导致的骨骼疾病的临床试验。 具体目的：1。通过组装大量患者的临床核心作为I和II期临床试验的候选者2。进行镰状细胞贫血和HBSC疾病的新型治疗试验2。进行创新的研究：镰状红细胞水合的遗传决定因素；内皮功能和G6PD缺乏症；新型口服剂的HBF诱导；改善为镰状细胞疾病儿童提供全面的预防服务；并且，通过维生素D和钙治疗改善骨骼健康3。为所有BCSCC患者提供高质量的临床护理，为患者提供教育和咨询，对医学专业人员的教育和研究培训以及血红蛋白病的基因测试4.将BCSCC的组成部分融合到凝聚力系统中，以使其与其他项目相互互动的互动效果，并使其互动越来越多地进行互动，并使其对互动的互动，并使其对其进行了反应，并将其置于互动，并使其对民族的疾病进行了反应。