Boston Comprehensive Sickle Cell Center

新千年开启了一个时代的非凡，对美国的镰状细胞病患者的承诺。有效的治疗方法现在是可用的，更长的，富有成效的生活与证明节省成本是可能的;新的治疗方法正在进行临床试验;了解生物学，疾病是由血管生物学和人类基因组的新见解充满活力。为了迎接挑战并抓住镰状细胞病研究的机遇，波士顿镰状细胞中心将开展镰状细胞贫血和HbSC病的新型治疗试验，重点是诱导HbF和预防镰状红细胞脱水。更多的基础研究将集中在红细胞生物学上，包括阳离子转运和缺氧和氧化对正常和镰状红细胞Gardos通道的影响以及镰状红细胞的粘附特性的研究。一氧化氮在镰状细胞病病理生物学中的作用将通过定义NO在镰状细胞转基因小鼠的肺和血管反应中的作用来研究。我们还将：建立一个临床核心，为治疗试验奠定基础;为患者提供教育和咨询， 医疗专业人员;提供血红蛋白病检测的国家资源。波士顿综合镰状细胞中心的所有组成部分将合并成一个有凝聚力的单位，以提高其有效性，超越其组成部分的总和，并鼓励与其他地方和国家镰状细胞中心的互动。

The new millennium opens an era of extraordinary, promise for sickle cell disease patients in the United States. Effective therapies are now available and longer, productive lives with proven cost savings are possible; novel treatments are undergoing clinical trials; understanding the biology, of the disease is being energized by new insights into vascular biology and the human genome. To meet the challenges and seize the opportunities of sickle cell disease research, the Boston Sickle Cell Center will carry out novel therapeutic trials in sickle cell anemia and HbSC disease focusing on the induction of HbF and prevention of dehydration of sickle erythrocytes. More basic studies will focus on erythrocyte biology and include studies of cation transport and the effect of hypoxlia and oxidation on the Gardos channel of both normal and sickle erythrocytes and adhesive properties of sickle erythrocytes. The role of nitric oxide in the pathobiology of sickle cell disease will be examined by defining the role of NO in the pulmonary and vascular responses in a sickle cell transgenic mouse. We will also: build a clinical core that will form a foundation for therapeutic trials; provide education and counseling for patients and medical professionals; provide a national resource for hemoglobinopathy testing. All components of the Boston Comprehensive Sickle Cell Center will be melded into a cohesive unit to increase its effectiveness beyond the sum of its components and encourage interactions with other local and national sickle cell centers.