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Boston Sickle Cell Research Consortium

波士顿镰状细胞研究联盟

基本信息

批准号：
7227738
负责人：
Lillian E McMahon
金额：
$ 15.49万
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/7227738
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 Professional Hemoglobin Hemoglobinopathies Hospitals Individual 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 United States National Institutes of Health Ursidae Family Woman abstracting experience follow-up hydroxyurea inhaled nitric oxide innovation multidisciplinary programs prospective repository sickle cell crisis sickling symposium tool

项目摘要

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）：