Tenth Cooley's Anemia Symposium: Basic Science, Clinical Research, and Patient Healthcare for Thalassemia Syndromes.

第十届库利贫血研讨会：地中海贫血综合征的基础科学、临床研究和患者保健。

• 批准号: 8986370
• 负责人: ELLIS J NEUFELD
• 金额: $ 2.2万
• 依托单位: NEW YORK ACADEMY OF SCIENCES
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-08-01 至 2016-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8986370
• 关键词: Academia; Academy; Adult; Adverse effects; Affect; Anemia; Area; Award; Basic Science; Blood Transfusion; Carbon Dioxide; Cardiac; Caregivers; Caring; Chelating Agents; Chicago; Child; Child Care; Childhood; Chronic; Clinical; Clinical Data; Clinical Management; Clinical Research; Clinical Trials; Collaborations; Communities; Community Healthcare; Comorbidity; Consensus; Cooley' s anemia; Data; Deformity; Development; Diabetes Mellitus; Diagnosis; Diagnostic; Digestive System Disorders; Disabled Persons; Disease; Drug Industry; Endocrine system; Erythrocytes; Event; Faculty; Fellowship; Fostering; Foundations; Funding Agency; Future; Gene Expression Regulation; Genes; Genetic; Globin; Goals; Government; Growth; Hares; Health; Health Personnel; Health Professional; Health Services Research; Healthcare; Heart; Hematological Disease; Hematology; Hematopoietic Stem Cell Transplantation; Hemoglobin; Hemoglobinopathies; Hepatic; Human; Image; Industry; Inherited; Institutes; International; Iron; Iron Overload; Joints; Kidney; Kidney Diseases; Knowledge; Life; Liver; Lung; Malignant Neoplasms; Medical; Medical Device; Medicine; Mendelian disorder; Meta-Analysis; Metabolism; Minority; Minority Groups; Mission; Molecular; Molecular Genetics; Molecular and Cellular Biology; Monitor; Mutation; National Heart, Lung, and Blood Institute; Nephrology; New York; Oral; Organ; Oxygen; Parents; Participant; Pathology; Pathway interactions; Patients; Pediatrics; Pharmaceutical Preparations; Pharmacologic Substance; Physicians; Policy Maker; Prevention; Process; Proteins; Puberty; Publications; Publishing; Quality of life; Radiology Specialty; Recommendation; Regulation; Research; Research Personnel; Resources; Rest; Role; Safety; Science; Scientist; Specialist; Stem cell transplant; Support Groups; Symptoms; Syndrome; Technical Expertise; Techniques; Technology; Thalassemia; Therapeutic; Time; Transfusion; Translational Research; Translations; Travel; United States; Woman; Work; abstracting; beta Globin; career; data sharing; dissemination research; drug development; experience; family genetics; fetal; fighting; gene therapy; hepcidin; improved; iron metabolism; knowledge translation; meetings; novel; posters; public health relevance; repository; stem cell biology; symposium; therapeutic target; tool; trait; wasting

 DESCRIPTION (provided by applicant): Tenth Cooley's Anemia Symposium - Thalassemia syndromes affect at least two million people worldwide, and researchers believe that several hundred million people may be "silent carriers" of a thalassemia trait, making it one of the most common single-gene disorders in humans. The Tenth Cooley's Anemia Symposium, to be held at Loews Chicago O'Hare Hotel October 18-22, 2015, represents the largest global gathering of thalassemia experts, and provides one of the few instances each decade that the international research community, the pharmaceutical industry, and funding agencies dedicated to fighting this blood disorder come together. This 3.5-day conference will convene approximately 350 attendees, including scientific researchers working in the fields of molecular and cellular biology stem cell biology, and genetics, as well as clinical specialists in hematology, hematology, nephrology, pathology, radiology, stem cell transplantation, transfusion medicine, and pediatrics, among other fields. The main objectives of this conference are to: (i) Provide a neutral forum for discussion of existing clinical data, emerging strategies, and future directions for research and treatment of genetic hemoglobinopathies, including Cooley's anemia; (ii) Attract/showcase early career and underrepresented investigators (including women, ethnic/racial minorities, and persons with disabilities) via short talks, posters, travel fellowshis, and to provide opportunities to interact with senior investigators; (iii) Disseminate the conferenc proceedings to the international community; and (iv) Foster collaboration between academia, medicine, industry, and government to promote knowledge exchange and successful translation of research into improved diagnostics and patient therapies for thalassemia and treatment- related organ damage. Cutting-edge topics to be discussed during this symposium include globin gene regulation, iron metabolism and hepcidin, management of thalassemia symptoms and iron overload, and preliminary results of clinical trials using gene therapy, stem cell transplantation, and novel drug pathways. The goals of this Symposium strongly support the mission of the National Heart, Lung, and Blood Institute (NHLBI) and National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in that the conference will focus on multiple priority areas of need: to advance hematology research and treatment of disorders of hemoglobin, and to improve safety and efficacy in blood transfusion medicine - including the diagnosis, management, and prevention of iron overload and subsequent damage to the kidney, liver, lungs, heart, and endocrine system. Discussions originating from the conference, and their dissemination via publication in a volume of Annals of the New York Academy of Sciences, will have global impact by improving scientific understanding, advancing breakthrough therapies, and ultimately reducing the healthcare burden of this family of genetic blood disorders.

 描述(申请人提供)：第十届库利贫血研讨会-地中海贫血综合征影响全球至少200万人，研究人员认为，数亿人可能是地中海贫血特征的“沉默携带者”，使其成为人类最常见的单基因疾病之一。第十届库利贫血研讨会将于2015年10月18日至22日在洛伊斯芝加哥奥黑尔酒店举行，是全球最大的地中海贫血专家会议，是每十年少数几个致力于抗击这种血液疾病的国际研究界、制药业和资助机构聚集在一起的例子之一。这次为期3.5天的会议将召集约350名与会者，包括分子和细胞生物学、干细胞生物学和遗传学领域的科学研究人员，以及血液学、血液学、肾脏病、病理学、放射学、干细胞移植、输血医学和儿科等领域的临床专家。这次会议的主要目的是：(I)提供一个中立的论坛，讨论包括库利氏贫血在内的遗传性血红蛋白疾病的研究和治疗的现有临床数据、新兴战略和未来方向；(Ii)通过简短的演讲、海报、旅行伙伴会议吸引/展示职业生涯早期和代表性不足的研究人员(包括妇女、少数民族/少数族裔和残疾人)，并提供与高级研究人员互动的机会；(Iii)向国际社会传播会议记录；以及(Iv)促进学术界、医学界、产业界和政府之间的合作，以促进知识交流，并成功地将研究转化为地中海贫血和治疗相关器官损伤的改进诊断和患者治疗。本次研讨会将讨论的前沿话题包括珠蛋白基因调控、铁代谢和海普西丁、地中海贫血症状和铁负荷的管理，以及使用基因治疗、干细胞移植和新药物途径的临床试验的初步结果。本次研讨会的目标大力支持国家心肺血液研究所(NHLBI)和国家糖尿病、消化及肾脏疾病研究所(NIDDK)的使命，会议将聚焦于多个优先需要的领域：推进血液学研究和血红蛋白疾病的治疗，提高输血医学的安全性和有效性-包括诊断、管理和预防铁超载以及随后对肾、肝、肺、心脏和内分泌系统的损害。这次会议产生的讨论，以及通过在《纽约科学院年鉴》上发表的讨论，将通过提高科学认识、推进突破性疗法并最终减轻这一遗传性血液疾病家族的医疗负担，产生全球影响。