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（由申请人提供）：第十届库利贫血研讨会 - 地中海贫血综合症影响着全世界至少 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 日在芝加哥奥黑尔洛斯酒店举行，这是全球最大规模的地中海贫血专家聚会，也是每十年国际研究界、制药行业和致力于对抗这种血液疾病的资助机构齐聚一堂的少数实例之一。此次为期3.5天的会议将聚集约350名与会者，包括分子和细胞生物学、干细胞生物学、遗传学领域的科研人员，以及血液学、血液学、肾病学、病理学、放射学、干细胞移植、输血医学、儿科等领域的临床专家。本次会议的主要目标是： (i) 提供一个中立的论坛，讨论遗传性血红蛋白病（包括库利贫血）的现有临床数据、新兴策略以及研究和治疗的未来方向； (ii) 通过简短的谈话、海报、旅行奖学金吸引/展示早期职业和代表性不足的调查员（包括妇女、少数族裔/种族和残疾人），并提供与高级调查员互动的机会； ㈢ 向国际社会传播会议记录； (iv) 促进学术界、医学界、工业界和政府之间的合作，以促进知识交流并将研究成功转化为改进的地中海贫血和治疗相关器官损伤的诊断和患者治疗。本次研讨会讨论的前沿话题包括珠蛋白基因调控、铁代谢和铁调素、地中海贫血症状和铁超载的治疗，以及使用基因治疗、干细胞移植和新型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、消化和肾脏疾病研究所 (NIDDK) 的使命，因为会议将重点关注多个优先领域的需求：推进血液学研究和血红蛋白疾病的治疗，并提高输血医学的安全性和有效性 - 包括铁的诊断、管理和预防 超负荷以及随后对肾脏、肝脏、肺、心脏和内分泌系统的损害。此次会议的讨论及其通过《纽约科学院年鉴》出版的传播，将通过提高科学理解、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而产生全球影响。