Boston Sickle Cell Research Consortium

波士顿镰状细胞研究联盟

• 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
• 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

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.

《基因组时代镰刀细胞治疗的新方向》会议在美国国立卫生研究院举行 2003年11月，美国卫生研究院公布。与会者的结论是：“这是一个有利的时机 承担基因组学的开发工具和方法，以开发出明显更有效的治疗方法 镰状细胞病“和”超过95%的受影响个人生活在美国以外， 基因组学在镰状细胞疾病上的应用需要全球视野和参与。 建议是“一个创新的多学科镰状细胞疾病研究网络， 应该建立表型良好的患者的中央预期登记，以及这种类型的特征 网络可能包括：DNA、细胞系、信使核糖核酸和血浆(后两者来自个体 当有症状和无症状时)，标准化表型，开放获取材料和数据， 纵向跟踪和临床试验数据库。 为了应对这些挑战，我们建议建立一个以波士顿医疗中心为核心的临床中心， 纽黑文和巴黎，法国。法国中心有近1500名特征良好的患者 之前曾与波士顿调查人员合作过。耶鲁中心将150名新的儿科患者带到 我们的临床核心。这两个中心都没有参与最近在美国进行的临床试验。 我们的研究课题包括所有国家的1-碱性血红蛋白基因分型和红细胞表型 镰状细胞病临床研究网络患者和将生物样本转移到存储库 供所有研究人员将来使用；1-吸入一氧化氮治疗镰状细胞血管闭塞的I/I11期试验 痛苦的插曲。