Eleventh Cooley's Anemia Symposium

第十一届库利氏贫血研讨会 项目总结 地中海贫血综合症影响着全球至少200万人，研究人员认为有几种 一亿人可能是地中海贫血的“沉默携带者”，这使它成为最常见的单纯性贫血之一。 人类的基因紊乱。第十一届库利氏贫血研讨会将在纽约举行 2022年10月17日至20日，美国纽约州约克城，代表着全球最大的地中海贫血专家聚会， 并提供了国际研究界每十年少数几个实例中的一个 制药行业和致力于抗击这种血液疾病的资助机构走到了一起。这个3.5- 一天的会议将召集大约250名与会者，其中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的专家，以及来自 血液学、干细胞移植、母婴健康、输血医学和儿科等领域， 还有其他的。这次会议的主要目标是：(1)提供一个中立的论坛，讨论 遗传性疾病研究和治疗的现有临床数据、新兴策略和未来方向 血红蛋白疾病，包括库利氏贫血；(2)吸引/展示早期职业生涯和代表性不足 调查人员(包括妇女、少数族裔/少数族裔和残疾人)通过简短的谈话、海报、 旅行研究金，并提供与高级调查人员互动的机会；(3)传播 向国际社会介绍会议记录；和(4)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 改进地中海贫血和与治疗相关的器官损伤的诊断和患者治疗。切割- 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、 地中海贫血的临床并发症的处理，包括关注全球主要的 健康问题，铁超载的评估和治疗，正在进行的基因治疗临床试验的结果， 造血干细胞移植，以及新药，以及基因治疗的创新方法。 本次研讨会的目标有力地支持了国家心肺血液研究所的使命和 国家糖尿病和消化及肾脏疾病研究所，会议将集中讨论多个优先事项 需要的领域：推进血液学研究、治疗和治疗血红蛋白疾病，并 提高输血医学的安全性和有效性-包括诊断、管理和 防止铁超载和随后对肝脏、心脏和内分泌系统的损害。讨论 源自会议，并通过出版在《纽约科学院年鉴》上传播 科学，将通过提高对科学的理解，推进突破，产生全球影响 治疗，并最终减轻这一遗传性血液疾病家族的医疗负担。

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.