2015 Lysosomal Disease Gordon Research Conference and Gordon Research Seminar

2015年溶酶体疾病戈登研究大会暨戈登研究研讨会

基本信息

批准号：
8830513
负责人：
Steven Upshaw Walkley
金额：
$ 1.5万
依托单位：
GORDON RESEARCH CONFERENCES
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-09-30 至 2015-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8830513
关键词：
Address Adopted Affect Alzheimer&apos s Disease Basic Science Biology Brain Brain Diseases Categories Cells Cessation of life Chronic Chronic Disease Clinical Clinical Trials Clinical Trials Design Combined Modality Therapy Communities Coupled Defect Development Discipline Disease Ensure Europe Event Exhibits Fertilization Functional disorder Funding Funding Agency Goals Health Hereditary Disease Homeostasis Incidence Individual Inherited Innovative Therapy International Investigational Therapies Italy Japan Knowledge Laboratories Lead Link Live Birth Lysosomes Molecular Nerve Degeneration Neurodegenerative Disorders Neurons Parkinson Disease Participant Pathogenesis Pathology Pathway interactions Patients Postdoctoral Fellow Proteins Renaissance Reporting Request for Applications Research Research Personnel Resort Role Running Scientist Source Staging Students System Texas Time Training United States National Institutes of Health Work abstracting base body system career cell type cost design disability effective therapy experience family genetics graduate student innovation insight interest link protein lysosomal proteins meetings nervous system disorder next generation novel posters preclinical study programs public health relevance success symposium therapy development

项目摘要

DESCRIPTION (provided by applicant): The Lysosomal Disease GRC had its first meeting at the Hotel Galvez in Galveston, Texas in 2011, and its second at the Renaissance Tuscany Il Ciocco Resort in Lucca (Barga), Italy in 2013. We are one of the newest of over 170 annual conferences organized by the GRC, an organization known world-wide for its high-quality, cutting-edge academic conferences, and indeed are the only new GRC approved as a repeat conference in their 2013 review. Lysosomal Disease represents a group of more than 50 disorders caused by inherited defects in a wide spectrum of proteins, many of which remain poorly understood. These disorders affect many organ systems, most notably brain, leading to chronic illness and death of affected individuals. The 2015 Lysosomal Disease GRC will again offer a critical venue for addressing major challenges involving lysosomal diseases, including the need for better understanding of (1) the role of the greater lysosomal system in maintaining cell homeostasis in health and disease, (2) lysosomal disease pathogenic mechanisms underlying the dysfunction of neurons and brain, as well as other cell types/organs/systems, (3) specific functions of lysosomal and non-lysosomal proteins, defects in which cause lysosomal disease, (4) the relationship between lysosomal dysfunction and other chronic neurodegenerative conditions (e.g., Alzheimer's and Parkinson's), and (5) the extent to which heterozygosity in autosomal and X-linked lysosomal diseases can lead to cellular and clinical disease. Additionally there is the challenge (6) through rigorous preclinical studies to develop new and innovative therapies for these disorders coupled with (7) initiating and completing effective clinical trials. To address these important topics we have to date invited, as speakers and discussants, 27 leading and internationally known scientists and clinicians working in the field of lysosomal disease. Each has accepted our invitation and will join what we anticipate to be an additional 170 attendees; fully one-third of whom we anticipate will be junior investigators, postdoctoral fellows and graduate students who are at the early stages of their careers. Importantly, the success of our 2011 meeting led to the GRC Organization selecting the Lysosomal Disease meeting as a host for a Gordon Research Seminar (GRS), a special meeting organized and run by graduate students and early postdoctoral fellows. Our 2015 meeting has again been selected to host a GRS. The presence of the GRS insures that the very best and the brightest of the next generation of lysosomal disease researchers will be part of the GRC as well. The discussion and cross fertilization of ideas and approaches occurring as part of the GRC and GRS meetings we believe will accelerate not only our understanding of the role of the lysosomal system in health and in disease, but also move us closer to effective therapies for many of these disorders.

描述（由申请人提供）：溶酶体疾病 GRC 于 2011 年在德克萨斯州加尔维斯顿的加尔维兹酒店举行了第一次会议，并于 2013 年在意大利卢卡（巴尔加）的万丽托斯卡纳 Il Ciocco 度假村举行了第二次会议。 GRC 组织的 170 多场年度会议中最新的一次，GRC 是一个以其高质量、前沿学术会议而闻名于世的组织，是唯一在 2013 年审查中被批准为重复会议的新 GRC 溶酶体疾病代表了由多种蛋白质的遗传缺陷引起的 50 多种疾病，其中许多疾病仍影响许多器官系统。最显着的是大脑，导致受影响个体的慢性疾病和死亡，2015 年溶酶体疾病 GRC 将再次为解决涉及溶酶体疾病的重大挑战提供一个重要场所，包括需要更好地了解 (1) 的作用。维持健康和疾病中细胞稳态的更大溶酶体系统，（2）神经元和大脑以及其他细胞类型/器官/系统功能障碍背后的溶酶体疾病致病机制，（3）溶酶体和非溶酶体的特定功能蛋白质、导致溶酶体疾病的缺陷，(4) 溶酶体功能障碍与其他慢性神经退行性疾病（例如阿尔茨海默病和帕金森病）之间的关系， (5) 常染色体和 X 连锁溶酶体疾病的杂合性可导致细胞和临床疾病的程度。此外，还存在挑战 (6) 通过严格的临床前研究来开发针对这些疾病的新的和创新的疗法 (7)。）启动并完成有效的临床试验。迄今为止，我们已邀请 27 位在溶酶体疾病领域工作的领先的国际知名科学家和信徒作为演讲者和讨论者。将加入我们预计的另外 170 名与会者；我们预计其中三分之一将是初级研究员；重要的是，我们 2011 年会议的成功导致 GRC 组织选择溶酶体疾病会议作为戈登研究研讨会 (GRS) 的主办方，这是一次组织和举办的特别会议。我们的 2015 年会议再次被选为 GRS 的主办方，GRS 的出席确保了下一代最优秀、最聪明的人才。溶酶体疾病研究人员也将成为 GRC 的一部分，我们相信，作为 GRC 和 GRS 会议的一部分，我们相信，这不仅会加速我们对溶酶体系统在健康和疾病中的作用的理解。，同时也让我们更接近针对许多此类疾病的有效疗法。