The Lysosomal Disease Network's 10th Annual WORLD Symposium

第十届世界学术研讨会将于2014年2月11日至13日在美国加利福尼亚州圣地亚哥举行。这个研究会议是一个多学科论坛，展示了溶酶体疾病的基础科学、转化研究和临床治疗的最新进展。会议的主题强调了主要目的，即评估将实验研究用于人类治疗的机制和障碍。其他目标建立在此基础上，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，从而提高对这些疾病的生化、免疫学、遗传学和临床表现的认识；3)识别和讨论诊断检测、筛查和治疗方面的最新发现；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，它们可能很少接触到在其当前感兴趣的领域之外的领域所做的工作。这次会议允许在溶酶体疾病方面分享知识和进展，并提供讨论治疗结果的机会。从多中心联盟“溶酶体疾病网络”（U54 NS065768，罕见病临床研究网络成员）的发展可以看出世界研讨会的协同性质。除研究人员和临床医生外，世界研讨会欢迎希望参加的患者，家长，护理人员和所有患者倡导者。这一独特的功能允许一个小的，地理上不同的患者群体获得有关溶酶体疾病最新科学进展的信息，并使科学家和临床医生能够前所未有地接触到诊所外的患者；提供一个论坛来共享信息，否则就不存在。该计划分为六个部分，从“基础研究”到“转化研究”再到“临床研究”。每一届会议，项目委员会将选出两名受邀演讲者，并从提交的摘要中填补剩余的项目（提交截止日期为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基础”，在研究会议之前的周一（2014年2月10日）。在前几年课程的基础上，本课程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小时扩展到一整天的教学，为构成研讨会内容的前沿研究报告提供一个共同的理解。为了促进高级和年轻研究人员之间的关系，我们将再次赞助年轻研究人员的旅行，促进他们参加“溶酶体101”和世界会议。指导新科学家是LDN和RDCRN的一个主要目标。

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.