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: 8793924
• 负责人: Chester B. Whitley
• 金额: $ 2万
• 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/8793924
• 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

DESCRIPTION (provided by applicant): 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 clinician's 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.

描述（由申请人提供）：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）。这次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临床治疗的最新进展。会议的主题强调了主要目的，即评估对人类治疗的基准研究的机制和障碍。其他目标是建立在此基础上，并实施溶酶体疾病网络（LDN）的目标：1）进一步发展LDN的机制和使命； 2）促进科学家之间的跨学科合作，从而提高了有关这些疾病的生化，免疫，遗传和临床表现的知识； 3）识别并讨论诊断测试，筛查和治疗中的最新发现； 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。由于许多LDN成分调查和/或治疗这些疾病的特定方面，因此在当前感兴趣的领域以外的地区所做的工作可能很少。这次会议允许共享溶酶体疾病的知识和进步，并提供了讨论治疗结果的机会。在多中心财团“溶酶体疾病网络”的增长中，可以看到世界研讨会协同性质的证据（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，世界研讨会还欢迎希望参加的患者，父母，看护人和所有希望参加​​的患者拥护者。这一独特的功能允许少量的，地理上差异的患者人群访问有关溶酶体疾病最新科学进步的信息，并为科学家和临床医生提供了前所未有的诊所诊所中患者的机会；提供一个论坛来共享否则不存在的信息。该计划分为六个课程，从“基础研究”延伸到“转化研究”到“临床研究”。对于每个会议，计划委员会将选择两名受邀发言人，并将从提交的摘要中填写该计划的其余部分（提交截止日期为2013年10月1日）。在研究会议之前（2014年2月10日）之前，该研讨会连续第三年将包括“溶酶体101，溶酶体生物学和疾病的基本原理”（2014年2月10日）。这次教育会议将建立在不取代前几年的课程中，将为与会者提供生物化学，免疫学，分子生物学和溶酶体疾病遗传学的基本概念。 2014年，溶酶体101将从4个小时扩展到一整天的教学，为构成研讨会内容的尖端研究演示提供了共同的理解。为了促进高级和年轻调查员之间的关系，我们将再次赞助年轻调查员旅行，促进他们参加“溶酶体101”和世界会议的参加。新科学家的指导是LDN和RDCRN的主要目标。