2019 Lysosomal Diseases Gordon Research Conference and Seminar

2019年溶酶体疾病戈登研究会议暨研讨会

• 批准号: 9760992
• 负责人: Beverly L. Davidson
• 金额: $ 1万
• 依托单位: GORDON RESEARCH CONFERENCES
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-03-01 至 2020-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9760992
• 关键词: Address; Advanced Development; Affect; Animals; Asia; Automobile Driving; Big Data; Biological Models; Biology; Calendar; Canada; Categories; Cessation of life; Chronic Disease; Clinical; Communities; Complement; Country; Data; Defect; Devices; Diagnosis; Discipline; Disease; Disease Outcome; Enzymes; Equilibrium; Europe; Faculty; Fees; Fertilization; Functional disorder; Funding; Genetic; Goals; Health; Human; Impairment; Individual; Industry; Inherited; Innovative Therapy; International; Knowledge; Laboratories; Link; Live Birth; Lysosomal Storage Diseases; Lysosomes; Metabolic Diseases; Methods; Minority; Molecular; Natural History; Organism; Participant; Pathogenesis; Pathogenicity; Pathologic; Pathology; Pathway interactions; Patients; Play; Postdoctoral Fellow; Prevalence; Process; Proteins; Rare Diseases; Request for Applications; Research; Research Personnel; Role; Science; Scientist; Source; System; Systems Biology; Texas; Training; Translations; United States National Institutes of Health; Woman; Work; base; body system; design; effective therapy; experience; graduate student; innovation; meetings; men; next generation; novel therapeutics; posters; premature; prevent; programs; research and development; small molecule; standard care; symposium; therapy development; tool

Project Summary/Abstract Lysosomal diseases represent more than 70 disorders caused by inherited defects in a spectrum of organellar proteins; they affect many organ systems progressively to cause chronic illness and premature death. The Lysosomal Disease Gordon Research Conference (GRC) has played a crucial role in stimulating discoveries in this field. The 2019 Lysosomal Disease GRC/Gordon Research Seminar (GRS), held at the Hotel Galvez in Galveston, Texas, will offer a critical venue for addressing major topics in lysosomal biology, disease mechanisms, diagnosis and therapy, including (i) lysosomal systems biology, where `big data' is opening the door to genetic and cellular systems at play in the pathophysiology of disease: (ii) innovations in methods that reverse, rather than prevent or stabilize, pathogenesis, a critical advance as many individuals with these disorders come to light long after the pathology is established: (iii) emerging tools to study lysosomal diseases, at the cellular level and in the whole animal; (iv) mechanisms of pathogenesis in disorders that have recently been linked to lysosomal dysfunction; (v) progress in our understanding of lysosome-resident channels, a field that is emerging as critical to ascertaining how various deficiencies impair lysosome function; (vi) lysosome biology; and (vii) advances in clinical approaches focused on lysosomal disease. This conference is unique in the academic calendar and an outstanding complement to other existing lysosomal disease forums in USA and Europe. To address these topics we have invited, as speakers and discussants, 42 scientists and clinicians working in the lysosomal disease and related fields. Importantly, there is a mix of junior and senior investigators from many countries, and we made every effort to include women and minorities. Additionally, we have formatted this year's conference for an additional 12 speakers to be selected from the abstracts, giving us an excellent opportunity to balance the program among junior and senior investigators, women and men, and to encourage minority participants. Of the current program presented in the application, 95% have accepted our invitation. Based on historical data for this conference, we anticipate that approximately one-third of attendees (aside from invited speakers and discussants) will be junior investigators, postdoctoral fellows and graduate students, and all together there will be approximately 160 people focused on lysosome disease, from the science underlying the field to the translation of those findings to patients suffering from lysosomal disease. Finally, the associated GRS insures that the very best and the brightest of the next generation of lysosomal disease researchers will also be integrated within the GRC community. The discussion and cross fertilization of ideas and approaches occurring as part of the GRC and GRS meetings will accelerate our understanding of the role of the lysosomal system in health and in disease and continue to advance the development of effective therapies.

项目概要/摘要 溶酶体疾病代表了由一系列细胞器遗传缺陷引起的 70 多种疾病 蛋白质；它们逐渐影响许多器官系统，导致慢性疾病和过早死亡。这 溶酶体疾病戈登研究会议 (GRC) 在刺激发现方面发挥了至关重要的作用 在这个领域。 2019年溶酶体疾病GRC/Gordon研究研讨会（GRS）在加尔维斯酒店举行 位于德克萨斯州加尔维斯顿的会议将为解决溶酶体生物学、疾病等主要主题提供一个重要场所 机制、诊断和治疗，包括（i）溶酶体系统生物学，其中“大数据”正在开放 遗传和细胞系统在疾病病理生理学中发挥作用的大门：（ii） 逆转而不是预防或稳定发病机制的方法，这是许多人的一项关键进步 患有这些疾病的个体在病理学确定很久之后才被发现：(iii) 新兴工具 在细胞水平和整个动物中研究溶酶体疾病； (iv) 发病机制 最近与溶酶体功能障碍有关的疾病； (v) 我们的理解取得进展 溶酶体驻留通道，这个新兴领域对于确定各种缺陷如何发挥关键作用 损害溶酶体功能； (vi) 溶酶体生物学； (vii) 临床方法的进展集中于 溶酶体疾病。这次会议在学术日历中是独一无二的，也是对 美国和欧洲其他现有的溶酶体疾病论坛。为了解决这些主题，我们邀请了 演讲者和讨论者包括 42 名从事溶酶体疾病及相关领域工作的科学家和临床医生。 重要的是，有来自许多国家的初级和高级研究人员，我们尽了一切努力 包括妇女和少数民族。此外，我们还为今年的会议额外安排了 12 场会议 从摘要中选出演讲者，这给了我们一个很好的机会来平衡该计划 初级和高级调查员，女性和男性，并鼓励少数群体参与者。目前的 申请中提出的计划，95% 接受了我们的邀请。根据这方面的历史数据 会议上，我们预计大约三分之一的与会者（除了受邀演讲者和 讨论者）将是初级研究员、博士后研究员和研究生，所有这些都将 大约 160 人关注溶酶体疾病，从该领域的基础科学到 将这些发现转化为患有溶酶体疾病的患者。最后，相关的 GRS 确保 下一代溶酶体疾病研究人员中最优秀和最聪明的人也将成为 融入 GRC 社区。思想和方法的讨论和交叉融合 作为 GRC 和 GRS 会议的一部分举行的会议将加速我们对溶酶体作用的理解 健康和疾病系统，并继续推进有效疗法的开发。