Boston Sickle Cell Research Consortium

波士顿镰状细胞研究联盟

• 批准号: 7065887
• 负责人: Lillian E McMahon
• 金额: $ 15.58万
• 依托单位: BOSTON MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-05-01 至 2011-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7065887
• 关键词: blood disorder chemotherapy; clinical research; clinical trials; cooperative study; drug screening /evaluation; erythrocytes; genetic registry /resource /referral center; genotype; hemoglobin; human subject; human therapy evaluation; nitric oxide; patient /disease registry; patient oriented research; sickle cell anemia; sickle cell crisis

DESCRIPTION (provided by applicant): The conference, "New Directions for Sickle Cell Therapy in the Genome Era" was held at the National Institutes of Health in November, 2003. The participants concluded that "the time is propitious to bring to bear the developing tools and approaches of genomics to develop markedly more effective therapies for sickle cell disease" and as "over 95% of affected individuals are living outside the United States, the application of genomics to sickle cell disease requires a global perspective and involvement." A key recommendation was that "An innovative multidisciplinary Sickle Cell Disease Research Network with a central prospective registry of well phenotyped patients should be established" and that "Features of such a network might include: Respositories of DNA, cell lines, mRNA, and plasma (the latter two from individuals when both symptomatic and asymptomatic), Standardized phenotypes, Open access to materials and data, Longitudinal follow-up and a Clinical trials database." To meet these challenges we propose to establish a clinical core encompassing Medical Centers in Boston, New Haven and Paris, France. The French Center has more that almost 1500 well characterized patients and has partnered before with Boston investigators. The Yale Center brings 150 new pediatric patients to our Clinical Core. Neither of these centers have participated in recent clinical trials in the United States. Our research topics include 1- basic hemoglobin genotyping and erythrocyte phenotyping of all National Sickle Cell Disease Clinical Research Network patients and the transfer of biological samples to a repository for future use by all investigators; 1-a phase I I/I 11 trial of inhaled nitric oxide for sickle cell vasoocclusive painful episode. (End of abstract)

描述（由申请人提供）： 该会议是“基因组时代的镰状细胞疗法新方向”，于2003年11月在美国国家卫生研究院举行。参与者得出的结论是，“时间是有时的，有时是有机基因组学的发展工具和方法，以显着开发出对镰状细胞疾病的更有效疗法，并且在全球疾病外的疾病中，疾病的疾病是疾病的疾病。参与。”一个关键建议是，“应建立具有良好表型患者的中央前瞻性注册表的创新性多学科细胞疾病研究网络，应建立“这种网络的特征”可能包括：DNA，细胞系，mRNA和等离子体的呼吸措施（在有症状和临时性的近距离访问材料和数据时，后两个人，后两个人），后两个，材料和数据，材料和数据，材料和数据，材料和数据，材料和数据，材料和数据，材料和材料，材料和数据。 数据库。” 为了应对这些挑战，我们建议在法国纽黑文和巴黎的波士顿和巴黎建立一个临床核心。法国中心的患者的特征性差不多1500名，并且与波士顿调查人员合作。耶鲁大学中心将150名新的儿科患者带入我们的临床核心。这些中心都没有参加过美国最近的临床试验。我们的研究主题包括1-所有国家镰状细胞疾病临床研究网络患者的基本血红蛋白基因分型和红细胞表型，以及将生物样品转移到所有研究人员将来使用的存储库中； 1-A I/I I/I 11次吸入一氧化氮的试验，用于镰状细胞血管判断性发作。 （抽象的结尾）