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.