The Lysosomal Disease Network's 10th Annual WORLD Symposium

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（美国加利福尼亚州圣地亚哥）举行。本研究 会议是一个多学科的论坛，介绍了基础科学，转化研究， 溶酶体疾病的临床治疗。会议的主题强调了首要目标，即 评估机制和障碍，将实验室研究用于人类治疗。其他目的建立 在此基础上，并实施溶酶体疾病网络（LDN）的目标，以：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， 提高有关这些疾病的生化、免疫、遗传和临床表现的知识， 疾病; 3）确定并讨论诊断测试，筛查和治疗的最新发现;以及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成分研究和/或治疗这些疾病的特定方面，它们可能很少暴露于 在他们目前感兴趣的领域之外所做的工作。这次会议允许分享知识， 在溶酶体疾病的进展，并提供了讨论治疗结果的机会。证据 从多中心联合体的发展中可以看出世界研讨会的协同作用 “溶酶体疾病网络”（U 54 NS 065768，罕见疾病临床研究网络成员）。 除了研究人员和临床医生，世界研讨会欢迎患者，父母，照顾者， 所有愿意参加的病人辩护律师这种独特的功能允许一个小的，地理上不同的病人， 人口获得有关溶酶体疾病的最新科学进展的信息，并为科学家 和临床医生前所未有地接触到诊所以外的病人;提供了一个分享的论坛 否则不存在的信息。该计划分为六个部分，从“基本”延伸到“基本”。 从“转化研究”到“临床研究”。“对于每一届会议，计划委员会将 选择两个受邀发言人，并将填补从提交的摘要（提交程序的其余部分 截止日期为2013年10月1日）。连续第三年，研讨会将包括一个教学部分， “溶酶体101，溶酶体生物学和疾病的基础”在研究前的星期一 会议（2014年2月10日）。在前几年课程的基础上， 教育会议将提供与会者的基本概念的生物化学，免疫学，分子 溶酶体疾病的生物学和遗传学。2014年，Lysosomes 101将从4小时扩展到一整天， 指导，为构成该课程的前沿研究报告提供了一个共同的理解。 专题讨论会内容。为了促进资深和年轻研究人员之间的关系，我们将一次 再次赞助年轻研究人员的旅行，促进他们参加“Lysosomes 101”和世界 会议新科学家的指导是LDN和RDCRN的主要目标。

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.