The Lysosomal Disease Network's 10th Annual WORLD Symposium

溶酶体疾病网络第十届年度世界研讨会

• 批准号: 8648085
• 负责人: Chester B. Whitley
• 金额: $ 1.5万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-30 至 2014-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8648085
• 关键词: Access to Information; Achievement; Address; Advocate; Area; Attention; Basic Science; Biochemical; Biochemistry; Biology; Caregivers; Clinic; Clinical; Clinical Research; Clinical Treatment; Clinical Trials; Collaborations; Diagnosis; Diagnostic tests; Disease; Disease Progression; Education; Educational Curriculum; Ethics; Evaluation; Event; Exposure to; Fostering; Funding; Genetic; Goals; Growth; Hour; Human; Immunologics; Immunology; Individual; Instruction; Intervention; Knowledge; Life; Lysosomes; Medical; Mentors; Metabolic; Metabolism; Mission; Modeling; Molecular Biology; Molecular Genetics; Natural History; Nature; Neonatal Screening; Parents; Patients; Physicians; Principal Investigator; Progress Reports; Public Policy; Rare Diseases; Reporting; Research; Research Peer Review; Research Personnel; Research Project Grants; Schools; Science Policy; Scientific Advances and Accomplishments; Scientist; Time; Translational Research; Travel; Treatment outcome; United States National Institutes of Health; Work; abstracting; base; cost; economic impact; improved; innovation; interdisciplinary collaboration; interest; lectures; meetings; member; multidisciplinary; nervous system disorder; patient population; programs; public health relevance; response; screening; success; symposium

Project Summary: The 10th annual WORLD Symposium will be held February 11-13, 2014 (San Diego, CA, USA). This research meeting is a multidisciplinary forum presenting the latest advances in basic science, translational research, and clinical treatments for lysosomal diseases. The theme of the meeting emphasizes the primary aim, which is to assess the mechanisms, and obstacles, for taking bench research into human therapy. Additional aims build upon this, and implement the goals of the Lysosomal Disease Network (LDN) to: 1) further develop the mechanisms and mission of the LDN; 2) foster interdisciplinary collaboration between scientists, leading to improved knowledge regarding the biochemical, immunologic, genetic, and clinical manifestations of these diseases; 3) identify and discuss the latest findings in diagnostic testing, screening, and treatment; and 4) identify areas that need more basic/clinical research, public policy, and regulatory attention. Because many LDN constituents investigate and/or treat specific aspects of these diseases, they may have little exposure to work done in areas outside their current area of interest. This meeting allows for the sharing of knowledge and advances across lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Evidence of the synergistic nature of the WORLD Symposium can be seen in the growth of the multi-center consortium "Lysosomal Disease Network" (U54 NS065768, a member of the Rare Diseases Clinical Research Network). In addition to researchers and clinicians, the WORLD Symposium welcomes patients, parents, caregivers, and all patient advocates who wish to attend. This unique feature allows a small, geographically-divergent patient population access to information on the latest scientific advances in lysosomal diseases and gives scientists and clinicians unprecedented access to patients outside the clinic setting; providing a forum for sharing information that otherwise does not exist. The program is organized into six sessions, extending from "basic research" through "translational research" to "clinical research." For each session, the Program Committee will select two invited speakers and will fill the remainder of the program from submitted abstracts (the submission deadline is October 1, 2013). For the third consecutive year, the Symposium will include a didactic component, "Lysosomes 101, Fundamentals of Lysosomal Biology and Disease" on the Monday preceding the research meeting (February 10, 2014). Building on-but not supplanting-the curriculum of the previous years, this educational session will offer attendees the basic concepts of the biochemistry, immunology, molecular biology, and genetics of lysosomal disease. In 2014, Lysosomes 101 will expand from 4 hours to a full day of instruction, providing a common understanding for the cutting-edge research presentations that constitute the Symposium content. In order to foster relationships between senior and young investigators, we will once again sponsor travel for young investigators, facilitating their attendance of "Lysosomes 101" and the WORLD conference. The mentoring of new scientists is a major goal of the LDN and the RDCRN.

项目摘要： 第10届年度世界研讨会将于2014年2月11日至13日举行（美国加利福尼亚州圣地亚哥）。这项研究 会议是一个多学科论坛，介绍了基础科学，翻译研究和 溶酶体疾病的临床治疗。会议的主题强调了主要目的，即 评估用于对人类治疗的基准研究的机制和障碍。其他目标构建 因此，并将溶酶体疾病网络（LDN）的目标实施到：1）进一步发展 LDN的机制和任务； 2）培养科学家之间的跨学科合作，导致 有关这些知识的知识，有关这些知识，免疫，免疫学，遗传和临床表现 疾病； 3）识别并讨论诊断测试，筛查和治疗中的最新发现；和4） 确定需要更多基础/临床研究，公共政策和监管关注的领域。因为很多 LDN成分调查和/或治疗这些疾病的特定方面，它们可能几乎没有接触 在目前感兴趣的领域以外的地区进行的工作。这次会议允许共享知识和 溶酶体疾病的进展，并提供了讨论治疗结果的机会。证据 在多中心财团的增长中可以看到世界研讨会的协同性质 “溶酶体疾病网络”（U54 NS065768，稀有疾病临床研究网络的成员）。 除研究人员和临床医生外，世界研讨会还欢迎患者，父母，看护人和 所有希望参加​​的患者拥护者。这种独特的功能允许一个小的，地理上发散的患者 人口访问有关溶酶体疾病最新科学进步的信息，并为科学家提供 临床医生前所未有的临床患者访问诊所环境之外的患者；提供一个分享论坛 否则不存在的信息。该程序分为六个会议，从“基本 研究“通过“转化研究”到“临床研究”。对于每个会议，计划委员会将 选择两个邀请的演讲者，并将从提交的摘要中填充程序的其余部分（提交 截止日期是2013年10月1日）。在连续第三年，研讨会将包括一个教学成分， 在研究之前 会议（2014年2月10日）。建立在不取代的未取代 - 前几年的课程中 教育会议将为与会者提供生物化学，免疫学，分子的基本概念 生物学和溶酶体疾病的遗传学。 2014年，溶酶体101将从4小时扩展到整天 指导，为构成尖端研究演示的共同理解提供了共同的理解 研讨会内容。为了建立高级和年轻调查员之间的关系，我们将一次 再次赞助年轻调查员旅行，促进他们的“溶酶体101”和世界的出席 会议。新科学家的指导是LDN和RDCRN的主要目标。