Northern California Comprehensive Sickle Cell Centers

北加州综合镰刀细胞中心(NCCSCC)的目标是：1)促进基础研究、临床科学、疾病的心理社会方面的血红蛋白病研究；并启动多中心合作研究项目2)为受影响的人群提供血红蛋白病检测、咨询、教育和护理。为了追求这些目标，我们在儿童医院奥克兰研究所(CHORI)和加州大学旧金山分校(UCSF)制定了相互依存的基础研究计划。这些基本项目与西湾(加州大学旧金山分校/旧金山综合医院)和东湾(奥克兰儿童医院)的核心临床研究项目相辅相成。这些计划提供血红蛋白病检测、咨询、教育和全面护理，以维持参与NCCSCC研究的700多名镰状细胞病(SCD)患者的稳定、特征良好的群体 项目。临床、基础和协作网络研究项目的目标是：a)启动11a期随机试验，以确定LY315920[抗PLA2抑制剂]是否可以安全地用于预防即将到来的急性冠脉综合征，并表征其防止损害的机制。B)确定无神经症状的成年SCD患者的神经认知和神经影像异常的程度。C)通过对生理过程的研究，了解导致红细胞膜磷脂不对称的机制，并确定其在SCD患者体内的意义。D)利用BAC构建镰状细胞小鼠 从贝宁、CAR、塞内加尔和沙特阿拉伯四种镰刀单倍型患者中分离出β-珠蛋白簇，并研究其病理生理学和流变学。E)我们网络项目的目的是验证一种假设，即在患有症状的SCD a的年轻成年人中，非清髓性预适应方案可以在从HLA相合的同胞捐赠者进行干细胞移植后诱导稳定的供者宿主造血嵌合体。

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.