Boston Sickle Cell Research Consortium

波士顿镰状细胞研究联盟

基本信息

批准号：
7414377
负责人：
Lillian E McMahon
金额：
$ 35.11万
依托单位：
BOSTON MEDICAL CENTER
依托单位国家：
美国
项目类别：
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/7414377
关键词：
Acute Adolescent Medicine Adult Affect Age Ally Arginine Butyrate Biological Blood Vessels Boston Cardiology Cell Line Cells Childhood Clinic Clinical Clinical Research Clinical Trials Clinical Trials Database Clinical Trials Network Comprehensive Health Care DNA Data Data Collection Databases Development Discipline of obstetrics Disease Disease Management Elderly Elements Enrollment Erythrocytes Future Genetic Genome Genomics Genotype Gynecology Health Health Professional Hemoglobin Hemoglobinopathies Hospitals Individual Institutes Institution Laboratories Life Medical center Messenger RNA Mississippi Neurology New England Newborn Infant Nitric Oxide Nurses Operative Surgical Procedures Ophthalmology Orthopedics Pain Parents Paris, France Participant Patient Care Patient Transfer Patients Pediatric Hospitals Pediatrics Phase Phenotype Physicians Plasma Population Pulmonology Recommendation Recruitment Activity Registries Research Research Activity Research Personnel Research Project Grants Sampling Sickle Cell Sickle Cell Anemia Specialist Specimen Thalassemia Time Training United States Ursidae Family Woman experience follow-up hydroxyurea inhaled nitric oxide innovation multidisciplinary programs prospective repository sickle cell crisis sickling symposium tool

Acute Adolescent Medicine Adult Affect Age Ally Arginine Butyrate Biological Blood Vessels Boston Cardiology Cell Line Cells Childhood Clinic Clinical Clinical Research Clinical Trials Clinical Trials Database Clinical Trials Network Comprehensive Health Care DNA Data Data Collection Databases Development Discipline of obstetrics Disease Disease Management Elderly Elements Enrollment Erythrocytes Future Genetic Genome Genomics Genotype Gynecology Health Health Professional Hemoglobin Hemoglobinopathies Hospitals Individual Institutes Institution Laboratories Life Medical center Messenger RNA Mississippi Neurology New England Newborn Infant Nitric Oxide Nurses Operative Surgical Procedures Ophthalmology Orthopedics Pain Parents Paris, France Participant Patient Care Patient Transfer Patients Pediatric Hospitals Pediatrics Phase Phenotype Physicians Plasma Population Pulmonology Recommendation Recruitment Activity Registries Research Research Activity Research Personnel Research Project Grants Sampling Sickle Cell Sickle Cell Anemia Specialist Specimen Thalassemia Time Training United States Ursidae Family Woman experience follow-up hydroxyurea inhaled nitric oxide innovation multidisciplinary programs prospective repository sickle cell crisis sickling symposium tool

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项目摘要

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/I11trial of inhaled nitric oxide for sickle cell vasoocclusive painful episode.

会议是“基因组时代的镰状细胞疗法的新方向” 卫生研究院于2003年11月。参与者得出的结论是：“现在是时候带来承担开发的工具和基因组学的方法，以开发出明显更有效的疗法镰状细胞疾病”和“超过95％的受影响的人都居住在美国以外的人，将基因组学应用于镰状细胞疾病需要全球视角和参与。建议是“具有创新的多学科镰状细胞疾病研究网络应建立良好表型患者的中央前瞻性注册处网络可能包括：DNA，细胞系，mRNA和等离子体的呼吸器（后两个个体的后两个当有症状和无症状）时，标准化表型，公开访问材料和数据时，纵向随访和临床试验数据库。” 为了应对这些挑战，我们建议在波士顿建立一个临床核心，纽黑文和法国巴黎。法国中心的患者有近1500名。并以前与波士顿调查人员合作。耶鲁市中心为150名新的儿科患者带来我们的临床核心。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-基本的血红蛋白基因分型和所有国家的红细胞表型镰状细胞疾病临床研究网络患者以及生物样品转移到存储库供所有调查人员使用； 1-A阶段I/I/I11试图吸入的一氧化氮用于镰状细胞痛苦的情节。