PRF 11th Anniversary Workshop on Progeria - "Hand in Hand: Basic and Clinica

PRF 11 周年早衰症研讨会 - “手拉手：基础与临床

• 批准号: 8530081
• 负责人: LESLIE B GORDON
• 金额: $ 1.35万
• 依托单位: PROGERIA RESEARCH FOUNDATION, INC.
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-04-01 至 2014-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8530081
• 关键词: 13 year old; Aging; American Heart Association; Anniversary; Atherosclerosis; Basic Science; Bone Diseases; Boston; Cardiovascular Diseases; Cause of Death; Child; Clinical; Clinical Research; Clinical Sciences; Clinical Treatment; Clinical Trials; Collaborations; Disease; Doctor of Philosophy; Educational process of instructing; Educational workshop; Elements; Environment; Farnesyl Transferase Inhibitor; Finding by Cause; Fostering; Foundations; France; Funding; Gene Mutation; Genetic; Goals; Health; Heart Diseases; Indium; International; Interview; Investigation; Lamins; Lead; Life; Malignant Neoplasms; Mission; Myocardial Infarction; Outcome; Peer Review; Policies; Premature aging syndrome; Principal Investigator; Process; Progeria; Publications; Research; Research Personnel; Science; Scientist; Series; Sirolimus; Spain; Splice-Site Mutation; Staging; Stem Cell Research; Stroke; Syndrome; United States National Institutes of Health; Washington; age related; bisphosphonate; continuing medical education; drug discovery; experience; gene therapy; induced pluripotent stem cell; meetings; posters; premature; programs; public health relevance; success; telomere; tool; translational medicine; treatment trial

DESCRIPTION (provided by applicant): Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) is a rare, fatal segmental premature aging disease caused by a splice site mutation in LMNA. All children with Progeria die from heart attacks or strokes at an average age of 13 years following premature, progressive atherosclerosis. The mission of The Progeria Research Foundation (PRF) is to find the cause, treatments and cure for Progeria and its aging-related disorders. The 2013 PRF International Scientific Workshop on Progeria will be held in Bethesda, MD on April 24-26, 2013. This will be the sixth in a perpetual series conducted by PRF since 2001. All prior meetings were co-funded by the National Institutes of Health. With record-high peer-reviewed publications on Progeria in 2011 and 2012, there is a tremendous amount of new scientific information to discuss. Meeting Objectives: to create an ideal environment for collaborative discussion between basic and clinical scientists about how their collective experience with Progeria can accelerate the scientific progress in the fields of Progeria, cardiovascular disease (CVD) and aging. Program and Speakers: The meeting starts with an inspiring interview session with children living with Progeria. This is followed by Plenary Session speaker Francis Collins, MD, PhD, who discovered the Progeria gene mutation. He will focus on Progeria as a translational medicine success story which brings us unique keys to understanding elements of cardiovascular disease and aging. In a session entitled "Clinical Treatment Trials", trial principal investigators will present major findings. Mark Kieran, MD, PhD (Dana Farber Cancer Inst., Boston) and Nicolas Levy, MD (Marseilles, France) will discuss farnesyltransferase inhibitor, bisphosphonate and statin use in Progeria. Clinical experts on CVD and bone disease of Progeria and aging will also present. George Martin, MD (U. Washington), a top expert in the genetics of aging, will lead the session entitled "Aging, Cardiovascular Disease, and Progeria", which will include investigations on the connections between progerin and telomeres. Carlos L¿pez-Ot¿n, PhD (Oviedo, Spain) will moderate a session on progerin and lamin partners in health and disease, with leading lamin biologists such as Robert Goldman, PhD (Northwestern U.). Thomas Misteli, PhD (NCI) will lead a session on cutting edge strategies for research and discovery, including induced pluripotent stem cell research, genetic therapies, rapamycin and drug discovery. Two short-talk panel sessions will foster young investigators. Each talk will be accompanied by a poster with study details. Judith Campisi, PhD (Buck Inst.), a world-renowned aging investigator will moderate a basic science panel; Monica Kleinman, MD (Boston Children's Hosp.), who has led clinical policy panels for the American Heart Association and co-chairs Progeria clinical trials, will moderate a clinical science panel. We estimate 155 attendees, 39 speakers and 50 poster presentations. Educational tools include a poster competition and continuing medical education credits. Conclusion: Science presented at this meeting represents the next wave of discovery in Progeria and its relationships to CVD and aging.

描述（由申请人提供）：Hutchinson-Gilford早衰综合征（HGPS或早衰症）是一种罕见的、致命的节段性早衰疾病，由LMNA中的剪接位点突变引起。所有患有早衰症的儿童死于心脏病发作或中风，平均年龄为13岁，死于过早的进行性动脉粥样硬化。早老症研究基金会（PRF）的使命是寻找早老症及其衰老相关疾病的病因、治疗方法和治愈方法。2013年PRF国际早衰症科学研讨会将于2013年4月24日至26日在马里兰州贝塞斯达举行。这将是该基金会自2001年以来进行的一系列永久性活动中的第六次。之前的所有会议都由美国国立卫生研究院共同资助。随着2011年和2012年关于早衰症的同行评审出版物创纪录，有大量新的科学信息需要讨论。会议目标：为基础和临床科学家之间的合作讨论创造一个理想的环境，讨论他们与早衰症的集体经验如何加速早衰症，心血管疾病（CVD）和衰老领域的科学进步。节目和发言人：会议开始与儿童生活与早衰症鼓舞人心的采访会议。其次是全体会议 演讲者弗朗西斯柯林斯，医学博士，博士，谁发现了早衰症基因突变。他将专注于早衰症作为一个转化医学的成功故事，为我们带来了了解心血管疾病和衰老的独特关键。在题为“临床治疗试验”的会议上，试验主要研究者将介绍主要发现。Mark Kieran，MD，PhD（Dana Farber Cancer Inst.，Boston）和Nicolas Levy，MD（马赛，法国）将讨论法尼基转移酶抑制剂、双膦酸盐和他汀类药物在早衰症中的应用。心血管疾病和骨质疏松症和衰老的临床专家也将出席。乔治马丁，医学博士（美国）。华盛顿），一位衰老遗传学方面的顶级专家，将主持题为“衰老、心血管疾病和早衰症”的会议，其中将包括对早老蛋白和端粒之间联系的调查。卡洛斯L？佩什奥特n，博士（奥维耶多，西班牙）将主持一个会议上早老蛋白和核纤层蛋白的合作伙伴在健康和疾病，与领先的核纤层蛋白生物学家，如罗伯特戈德曼，博士（西北大学）。托马斯Misteli博士（NCI）将领导一个关于研究和发现的前沿战略的会议，包括诱导多能干细胞研究，遗传疗法，雷帕霉素和药物发现。两个简短的小组讨论会将培养年轻的调查人员。每个讲座将附有一张海报，介绍研究细节。Judith Campisi，博士（巴克研究所），一位世界著名的老龄化研究者将主持一个基础科学小组;莫妮卡·克莱曼，医学博士（波士顿儿童医院），他曾领导美国心脏协会的临床政策小组，并担任早衰症临床试验的联合主席，他将主持一个临床科学小组。我们估计有155名与会者，39名演讲者和50个海报演示。教育工具包括海报比赛和继续医学教育学分。结论：在这次会议上提出的科学代表了下一波发现早衰症及其与CVD和衰老的关系。