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Northern California Comprehensive Sickle Cell Centers

北加州综合镰状细胞中心

基本信息

批准号：
7995636
负责人：
ELLIOTT P. VICHINKSY
金额：
$ 1.26万
依托单位：
CHILDREN'S HOSPITAL & RES CTR AT OAKLAND
依托单位国家：
美国
项目类别：
财政年份：
2003
资助国家：
美国
起止时间：
2003-07-01 至 2010-03-31
项目状态：
已结题

项目摘要

The objectives of the Northern California Comprehensive Sickle Cell Center (NCCSCC) are:1) to facilitate hemoglobinopathy research in the areas of basic research, clinical science, psychosocial aspects of the disease; and to initiate multicenter collaborative research projects 2) to provide hemoglobinopathy detection, counseling, education and care to affected populations. To pursue these objectives, we have developed interdependent basic research programs at Childrens' Hospital Oakland Research Institute (CHORI) and University of California at San Francisco (UCSF). These basic programs are complemented by core clinical research programs in the West Bay (UCSF/San Francisco General Hospital) and in the East Bay (Childrens' Hospital Oakland]. These programs provide hemoglobinopathy detection, counseling, education and comprehensive care needed to maintain a stable, well characterized group of over 700 patients with sickle cell disease (SCD) who participate in NCCSCC research projects. Clinical, basic and collaborative network research projects aims are: A) To initiate a phase lla, randomized, trial to determine if LY315920 [anti PLA 2 inhibitor] can be safely used to prevent impending ACS and to characterize the mechanisms by which it prevents damage. B) To determine the extent of neurocognitve and neuromimaging abnormalities in neurologically asymtomatic adult SCD patients. C) To understand the mechanisms that result in phospholipid asymmetry in red cell membranes and to determine their in vivo significance in SCD patients by studying physiological processes. D) To construct sickle cell mice utilizing BAC's to isolate the beta-globin clusters from patients with the four sickle haplotypes: Benin, CAR, Senegal and Saudi Arabia, and to study their pathophysiology and rheology. E) Our network project's aim is to test hypothesis that a non-myeloablative conditioning regimen in young adults with symptomatic SCD a can induce stable donor host hematopoietic chimerism after stem cell transplantation from HLA identical sibling donors.

北加州综合镰状细胞中心 (NCCSCC) 的目标是：1) 促进血红蛋白病在基础研究、临床科学、疾病心理社会方面的研究；并启动多中心合作研究项目 2) 为受影响人群提供血红蛋白病检测、咨询、教育和护理。为了实现这些目标，我们在奥克兰儿童医院研究所 (CHORI) 和加州大学旧金山分校 (UCSF) 制定了相互依赖的基础研究项目。这些基础项目得到西湾（UCSF/旧金山总医院）和东湾（奥克兰儿童医院）核心临床研究项目的补充。这些项目提供血红蛋白病检测、咨询、教育和综合护理，以维持 700 多名参与 NCCSCC 研究的镰状细胞病 (SCD) 患者的稳定、特征明确的群体项目。临床、基础和协作网络研究项目的目标是： A) 启动 IIa 期随机试验，以确定 LY315920 [抗 PLA 2 抑制剂] 是否可以安全地用于预防即将发生的 ACS 并表征其预防损害的机制。 B) 确定无神经症状的成年 SCD 患者的神经认知和神经成像异常程度。 C) 了解导致红细胞膜磷脂不对称的机制，并通过研究生理过程来确定其在 SCD 患者体内的意义。 D) 利用 BAC 构建镰状细胞小鼠从具有四种镰状单倍型（贝宁、CAR、塞内加尔和沙特阿拉伯）的患者中分离出 β-珠蛋白簇，并研究其病理生理学和流变学。 E) 我们的网络项目的目的是检验这样的假设：对有症状的 SCD a 的年轻人来说，非清髓性预处理方案可以在 HLA 相同同胞供体的干细胞移植后诱导稳定的供体宿主造血嵌合。