2013 Lysosomal Disease Gordon Research Conference and Gordon Research Seminar

2013年溶酶体疾病戈登研究会议暨戈登研究研讨会

• 批准号: 8526613
• 负责人: Steven Upshaw Walkley
• 金额: $ 1.75万
• 依托单位: GORDON RESEARCH CONFERENCES
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-03-01 至 2014-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8526613
• 关键词: Address; Affect; Alzheimer' s Disease; Basic Science; Biological Markers; 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; Endocytosis; Ensure; Europe; Event; Exhibits; Faculty; Fertilization; Functional disorder; Funding; Funding Agency; Goals; Health; Hereditary Disease; Incidence; Individual; Inherited; Innovative Therapy; International; Investigational Therapies; Italy; Knowledge; Laboratories; Lead; Link; Live Birth; Lysosomes; Molecular; Molecular Genetics; Nerve Degeneration; Neurodegenerative Disorders; Neurons; Parkinson Disease; Participant; Pathogenesis; Pathology; Pathway interactions; Patients; Postdoctoral Fellow; Proteins; Renaissance; Request for Applications; Research; Research Personnel; Resort; Role; Running; Scientist; Site; Source; Staging; Students; System; Texas; Therapy Clinical Trials; Time; Training; United States National Institutes of Health; Validation; Work; abstracting; base; body system; career; cell type; cost; design; disability; effective therapy; enzyme replacement therapy; experience; family genetics; graduate student; innovation; interest; meetings; nervous system disorder; next generation; novel; posters; programs; public health relevance; success; symposium; therapy development; trafficking

DESCRIPTION (provided by applicant): Following from its highly successful inaugural meeting at the Hotel Galvez in Galveston, Texas, in 2011, this second meeting of the Gordon Research Conference (GRC) on Lysosomal Disease will take place at the Renaissance Tuscany Il Ciocco Resort in Lucca (Barga), Italy in April, 2013. The Lysosomal Disease GRC is 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. The Lysosomal Disease GRC evolved from the longstanding and highly successful Lysosomes and Endocytosis GRC, which for many years has focused principally on the burgeoning field of intracellular trafficking and endocytic mechanisms, rather than lysosomal function/disease. Yet 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 Lysosomal Disease GRC offers a critical venue for addressing the major challenges involving lysosomal diseases, including the need for better understanding of (1) pathogenic mechanisms underlying the dysfunction of neurons and brain, as well as other cell types/organs/systems, (2) specific functions of non-enzymatic, enigmatic proteins (NPC1 and NPC2, CLN3, TRPML1, others) causing some of the most difficult to treat lysosomal disorders and (3) the relationship between specific lysosomal diseases and other chronic neurodegenerative conditions (e.g., Niemann-Pick type C and Alzheimer's, Gaucher and Parkinson's, and so forth). Additionally there is the challenge to develop (4) new and innovative therapies for these disorders coupled with (5) appropriate biomarker identification and validation, with this followed by (6) effective clinical trial development. To address these important topics we have invited 41 leading and internationally known scientists and clinicians working in the field of lysosomal disease to join with approximately another 160 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 the 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. The presence of the GRS insures that the 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）：继 2011 年在德克萨斯州加尔维斯顿加尔维兹酒店举行的首次会议取得巨大成功之后，溶酶体疾病戈登研究会议 (GRC) 的第二次会议将在托斯卡纳 Il Ciocco 万丽度假酒店举行2013 年 4 月在意大利卢卡（巴尔加）举行。溶酶体疾病 GRC 是溶酶体疾病 GRC 组织的 170 多个年度会议中最新的会议之一GRC，一个以其高质量、前沿学术会议而闻名于世的组织。溶酶体疾病 GRC 是从长期存在且非常成功的溶酶体和内吞作用 GRC 演变而来的，多年来，该 GRC 主要关注细胞内运输和内吞机制的新兴领域，而不是溶酶体功能/疾病。然而，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，其中许多疾病仍然知之甚少。这些疾病影响许多器官系统，尤其是大脑，导致受影响个体的慢性疾病和死亡。溶酶体疾病 GRC 为解决涉及溶酶体疾病的主要挑战提供了一个关键场所，包括需要更好地了解 (1) 神经元和大脑以及其他细胞类型/器官/系统功能障碍的致病机制，(2 ) 非酶促、神秘的蛋白质（NPC1 和 NPC2、CLN3、TRPML1 等）的特定功能导致一些最难治疗的溶酶体疾病，以及 (3) 特定功能之间的关系溶酶体疾病和其他慢性神经退行性疾病（例如尼曼-匹克 C 型和阿尔茨海默病、戈谢病和帕金森病等）。此外，还面临着开发（4）针对这些疾病的创新疗法以及（5）适当的生物标志物识别和验证，以及（6）有效的临床试验开发的挑战。为了讨论这些重要主题，我们邀请了 41 位在溶酶体疾病领域工作的领先国际知名科学家和临床医生，以及大约另外 160 名与会者，我们预计其中三分之一将是初级研究员、博士后研究员和研究生。正处于职业生涯的早期阶段。重要的是，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，这是一次由研究生和早期博士后研究员组织和主持的特别会议。 GRS 的存在确保了下一代溶酶体疾病研究人员中最优秀、最聪明的人也将成为 GRC 的一部分。作为 GRC 和 GRS 会议一部分的讨论和思想和方法的交叉融合，我们相信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，而且还会使我们更接近针对许多疾病的有效疗法。这些疾病。