Boston Comprehensive Sickle Cell Center

新千年为美国镰状细胞病患者开启了一个非凡、充满希望的时代。 有效的治疗方法现已问世，而且有可能延长有效寿命，节约成本；新奇 治疗方法正在进行临床试验；对这种疾病的生物学的理解正在被新的 对血管生物学和人类基因组的洞察。迎接挑战，抓住机遇 ：细胞疾病研究，波士顿镰状细胞中心将对镰状细胞性贫血进行新的治疗试验 HBSC病，主要表现为诱导HbF和防止镰状红细胞脱水。更多 基础研究将集中在ED的红细胞生物学上，包括阳离子转运和低氧的影响的研究 正常和镰刀状红细胞Gardos通道的氧化和镰刀状红细胞的粘附性 红血球。一氧化氮在镰状细胞病的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肺和血管反应中的作用我们还将：建立一个 临床核心，将形成治疗试验的基础；为患者提供教育和咨询； ，医疗专业人员；为血红蛋白病检测提供国家资源。波士顿号的所有部件 综合镰刀细胞中心将融入一个有凝聚力的单元，以提高其效率，超越 总结其组成部分，并鼓励与其他地方和国家镰状细胞中心互动。

The new millennium opens an era of extraordinary, promise for sickle cell disease patients in the United States. Effective therapies are nov¿ 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 et31hrocytes. More basic studies will focus on eD'throcyte biology and include studies of cation transport and the effect of hypoxla 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 pulmona_' 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.