Eleventh Cooley's Anemia Symposium

第十一届库利贫血研讨会

基本信息

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
项目类别：
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&apos 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

项目摘要

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概要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，有几种数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疾病之一人类基因疾病第十一届库利贫血研讨会将在纽约举行。 2022 年 10 月 17 日至 20 日，美国纽约州约克市将举行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为数不多的例子之一制药行业和致力于对抗这种血液疾病的资助机构齐心协力，这 3.5-。日会议将召集约 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临床专家血液学、干细胞移植、母婴健康、输血医学、儿科等领域，本次会议的主要目标是： (i) 提供一个中立的论坛来讨论遗传学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病，包括库利贫血；(ii) 吸引/展示早期职业生涯和代表性不足；通过简短的谈话、海报、旅行奖学金，并提供与高级研究人员互动的机会；向国际社会公布会议记录；以及 (iv) 促进学术界之间的合作，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。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、地中海贫血临床并发症的管理，包括重点关注全球主要的α地中海贫血健康问题、铁过载的评估和治疗、正在进行的基因治疗临床试验的结果、造血干细胞移植、新药物以及基因治疗的创新方法。本次研讨会的目标有力支持国家心肺血液研究所的使命和国家糖尿病、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、血红蛋白疾病的治疗和治愈，以及提高输血医学的安全性和有效性——包括诊断、管理和预防铁过载以及随后对肝脏、心脏和内分泌系统的损害。源于会议，并通过《纽约科学院年鉴》上的出版物进行传播科学将通过提高科学理解、推进突破而产生全球影响疗法，并最终减轻这一遗传性血液疾病家族的医疗负担。