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COMPREHENSIVE SICKLE CELL CENTER OF MANHATTAN

COMPREHENSIVE SICKLE CELL CENTER OF MANHATTAN
曼哈顿综合镰状细胞中心
批准号:
6183191
负责人:
SERGIO S PIOMELLI
金额:
$201.44万
依托单位国家:
美国
项目类别:
财政年份:
1983
资助国家:
美国
项目状态:
已结题
起止时间:
1983-04-01 至 2003-03-31

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项目成果

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中文摘要
翻译
曼哈顿综合镰刀细胞中心是由2个人组成的财团 医学院(哥伦比亚和Mt.西奈)和4家医院(长老会, 圣卢克--罗斯福、哈莱姆区和芒特山西奈半岛)。病人的数量 接受服务的人有900人,几乎是岛上所有的病人 患有镰状细胞病的曼哈顿。 中心的临床单元为您提供“最先进的”个性化服务 为患者提供临床护理,患者反过来又参与各种 研究项目。 该中心研究活动的一个主要目标是 该病的分子生物学研究进展 基因转移的目标是在动物体内和体外进行的, 血红蛋白转换的控制和转基因小鼠的建立 疾病的动物模型作为平行方法。这些病人是 通过DNA分析对其进行详细的遗传分类及其基因分型 与临床表现和血液学数据相关,以得出 可用于靶向治疗的预后因素。 该中心的另一个领域侧重于治疗:间歇性治疗的使用 丁酸盐疗法有望增加HGB F(已证明 低至4%的HGBF百分比是中心的另一个单位 防止中风)。另一种治疗方法是试验 口服铁络合剂用于慢性输血患者。的研究 神经心理损伤与体液研究相关 凝血与反映内皮细胞损伤的分子整合 用“体内”研究微循环。 该中心旨在为所有项目提供凝聚力和协同效应。中心 得到了管理和统计核心的帮助,我的一个分子 生物学和血液学实验室核心,并通过红细胞生成 实验室核心。
英文摘要
The Comprehensive Sickle Cell Center of Manhattan is a consortium of 2 medical schools (Columbia and Mt. Sinai) and 4 hospitals (Presbyterian, St. Luke's-Roosevelt, Harlem and Mt. Sinai). The population of patients served consists of 900 individuals, nearly all the patients in the island of Manhattan with sickle cell diseases. The clinical units of the Center provide "state-of-the art" personalized clinical care to the patients, who, in turn, participate in a variety of research projects. One main goal of research activities of the Center focuses on the molecular biology approach to the disease The gene transfer goal is pursued in animals and in vitro, with the control of hemoglobin switching and the creation of transgenic mice as an animal model of the disease as parallel approaches. The patients are genetically classified in detail by DNA analysis and their genotype correlated with clinical manifestation and hematological data to derive prognostic factors that could be used to target therapy. Another area of the Center focuses on therapy: the use of intermittent butyrate therapy offers the promise of increasing Hgb F (it has been shown by another unit of the Center that a HgbF percentage as low as >4% is protective against stroke). Another therapeutic approach is the trial of oral Fe chelator for the patients on chronic transfusion. Studies of neuropsychological damage are correlated with humoral studies of coagulation and of molecules reflecting endothelial damage and integrated with "in vivo" studies of microcirculation. The Center aims to provide cohesion and synergy to all project. The center is assisted by an administrative and statistical core, my a molecular biology and hematological laboratory core, and by an erythropoiesis laboratory core.
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