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Cooley的贫血研讨会 项目摘要 Thalassya综合症在全球至少有200万人，研究人员认为有几个 一亿人可能是丘脑性特征的“沉默携带者”，使其成为最常见的单一特征之一 人类的基因疾病。第十一Cooley的贫血研讨会将在新的 2022年10月17日至20日，美国纽约州约克市代表了塔阿氏贫血专家的最大全球聚会， 并提供了每十年的少数几个实例之一，国际研究界， 制药行业以及致力于与这种血液疾病作斗争的资金机构聚集在一起。这个3.5- 一日会议将方便地大约有250名参加者，包括在该领域工作的科学研究人员 分子和细胞生物学，基因疗法和干细胞发育以及来自的临床专家 血液学，干细胞移植，母体健康，输血医学和儿科领域， 等等。这次会议的主要目标是：（i）提供一个中立论坛，以讨论 现有的临床数据，新兴策略以及通用研究和治疗的未来方向 血红蛋白病，包括库利的贫血； （ii）吸引/展示早期职业和人数不足 调查人员（包括妇女，种族/种族少数民族和残疾人），通过简短的谈话，海报， 旅行奖学金，并提供与高级调查员互动的机会； （iii）传播 国际社会的会议诉讼； （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成功翻译研究 改善诊断和患者疗法的thal症和治疗相关器官损伤。切割- 在本次研讨会期间要讨论的边缘主题包括球蛋白基因调节，铁代谢， 丘脑贫血的临床并发症的管理，包括关注阿尔法塔阿氏贫血，这是一个主要的全球 健康问题，铁超载的评估和治疗，基因治疗的临床试验结果， 造血干细胞移植和新药物以及基因治疗的创新方法。 这次研讨会的目标强烈支持国家心脏，肺和血液研究所的使命以及 美国国家糖尿病与消化和肾脏疾病研究所，会议将重点关注多个优先事项 需要的领域：提高血液研究，治疗和治愈血红蛋白疾病的治疗，以及 提高输血医学的安全性和效率 - 包括诊断，管理和 预防铁超负荷以及随后对肝脏，心脏和内分泌系统的损害。讨论 源自会议，以及通过在纽约学院的纪事中出版的传播 科学，将通过改善科学理解，推动突破来产生全球影响 疗法，并最终减少了这个遗传性血液疾病家族的医疗保健烧伤。