Eleventh Cooley's Anemia Symposium

第十一届库利贫血研讨会

• 批准号: 10538276
• 负责人: Janet L Kwiatkowski
• 金额: $ 1万
• 依托单位: NEW YORK ACADEMY OF SCIENCES
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-01 至 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10538276
• 关键词: Academia; Academy; Adherence; Adult; Affect; Anemia; Area; Award; Basic Science; Blood Transfusion; Bone Diseases; Carbon Dioxide; Caregivers; Caring; Child; Child Care; Childhood; Chronic; Clinical; Clinical Data; Clinical Management; Clinical Trials; Collaborations; Communities; Cooley' s anemia; Data; Data Pooling; Deformity; Development; Diagnosis; Diagnostic; Disabled Persons; Disease; Drug Industry; Endocrine system; Erythrocytes; Event; Fellowship; Fertility; Fetal health; Fostering; Foundations; Functional disorder; Funding Agency; Future; Gene Expression Regulation; Genes; Genetic; Globin; Goals; Government; Growth; Health Personnel; Health Professional; Healthcare; Heart; Heart Diseases; Hematological Disease; Hematology; Hematopoietic Stem Cell Transplantation; Hemoglobin; Hemoglobinopathies; Human; Impairment; Industry; Inherited; International; Iron; Iron Chelation; Iron Overload; Joints; Kidney; Knowledge; Life; Liver; Lung; Malignant Neoplasms; Medical; Medical Device; Medicine; Mendelian disorder; Minority Groups; Mission; Molecular; Molecular and Cellular Biology; Mutation; National Heart, Lung, and Blood Institute; National Institute of Diabetes and Digestive and Kidney Diseases; New York; New York City; Oral; Organ; Oxygen; Parents; Participant; Patient Outcomes Assessments; Patients; Pediatrics; Persons; Pharmacologic Substance; Physicians; Policy Maker; Prevention; Process; Proteins; Publications; Publishing; Quality of life; Recommendation; Regulation; Research; Research Personnel; Resources; Rest; Safety; Science; Scientist; Source; Specialist; Stem Cell Development; Stem cell transplant; Support Groups; Syndrome; Technical Expertise; Technology; Thalassemia; Therapeutic; Time; Transfusion; Translational Research; Translations; Travel; United States; Woman; alpha thalassemia major; alpha thalassemia minor; alpha-Thalassemia; beta Globin; career; comorbidity; conventional therapy; curative treatments; data sharing; drug development; experience; family burden; family genetics; fetal; fighting; gene therapy; gene therapy clinical trial; global health; healthcare community; improved; innovation; iron metabolism; knowledge translation; meetings; minority investigator; new therapeutic target; novel; novel therapeutics; posters; racial and ethnic; reduce symptoms; side effect; symposium; therapeutic target; tool; transfusion medicine; treatment strategy; wasting

Eleventh Cooley’s Anemia Symposium PROJECT SUMMARY 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 Eleventh Cooley's Anemia Symposium, which will be held The Cure in New York City, NY, USA on October 17–20, 2022, 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 250 attendees, including scientific researchers working in the fields of molecular and cellular biology, gene therapy, and stem cell development, as well as clinical specialists from the fields of hematology, stem cell transplantation, maternal-fetal health, transfusion medicine, and pediatrics, among others. 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 fellowships, and to provide opportunities to interact with senior investigators; (iii) Disseminate the conference 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, management of clinical complications of thalassemia including a focus on alpha thalassemia, a major global health problem, assessment and treatment of iron overload, results of ongoing clinical trials of gene therapy, hematopoietic stem cell transplantation, and novel drugs, as well as innovative approaches to gene therapy. The goals of this Symposium strongly support the mission of the National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases in that the meeting will focus on multiple priority areas of need: to advance hematology research, treatment and cure 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 liver, heart, and endocrine system. Discussions originating from the conference, and their dissemination via publication in 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.

第十一届库利贫血研讨会 项目摘要 地中海贫血综合征影响全球至少200万人，研究人员认为， 一亿人可能是地中海贫血特征的“沉默携带者”，使其成为最常见的单一- 人类的基因紊乱第十一届库利贫血研讨会，这将在新的治疗 2022年10月17日至20日在美国纽约州约克市举行的地中海贫血专家会议是全球最大的地中海贫血专家会议， 并提供了每十年为数不多的国际研究界， 制药业和致力于对抗这种血液疾病的资助机构走到一起。这个3.5- 为期一天的会议将召集约250名与会者，包括在该领域工作的科学研究人员 分子和细胞生物学，基因治疗和干细胞开发，以及来自 血液学、干细胞移植、母胎健康、输血医学和儿科学领域， 还有其他的本次会议的主要目标是：（一）提供一个中立的论坛， 现有的临床数据，新兴的策略，以及遗传性疾病研究和治疗的未来方向 血红蛋白病，包括库利氏贫血;（ii）吸引/展示早期职业和代表性不足 调查人员（包括妇女、少数民族/种族和残疾人）通过简短的谈话、海报， 提供旅行研究金，并提供与高级调查员互动的机会; ㈣促进学术界， 医学、工业和政府促进知识交流和研究成果的成功转化 改进地中海贫血和治疗相关器官损伤的诊断和患者治疗。切割- 在这次研讨会上将讨论的边缘话题包括珠蛋白基因调控，铁代谢， 地中海贫血的临床并发症的管理，包括重点α地中海贫血，一个主要的全球 健康问题、铁超载评估和治疗、正在进行的基因治疗临床试验的结果 造血干细胞移植和新药，以及基因治疗的创新方法。 本次研讨会的目标大力支持国家心肺血液研究所的使命， 国家糖尿病、消化和肾脏疾病研究所表示，会议将重点关注多个优先事项， 需要的领域：推进血液学研究，治疗和治愈血红蛋白疾病， 提高输血医学的安全性和有效性-包括诊断、管理和 预防铁过载和随后对肝脏、心脏和内分泌系统的损害。讨论 源自会议，并通过纽约科学院年鉴的出版传播， 科学，将通过提高科学认识，推进突破， 治疗，并最终减轻这一家族遗传性血液疾病的医疗负担。