Lysosomal Disease Network-9th Annual WORLD Symposium

简介（由申请人提供）：第九届年度世界研讨会将于2013年2月13-15日在美国佛罗里达州奥兰多举行。这次研究会议是一个多学科论坛，介绍了溶酶体疾病的基础科学、转化研究和临床试验的最新进展。会议的主题将强调主要目标，即评估将实验研究用于人类治疗的机制和障碍。在此基础上，进一步实现溶酶体疾病网络（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，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成员调查和/或治疗这些疾病的特定方面，他们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。这次会议允许分享所有溶酶体疾病的知识和进展，并提供讨论治疗结果的机会。多中心联盟“溶酶体疾病网络”（U54 NS065768，罕见病临床研究网络，RDCRN的成员）的发展证明了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，否则不存在。该计划分为六个阶段，从“基础研究”到“转化研究”再到“临床研究”。对于每个人，项目委员会将选择两名受邀演讲者，并将从提交的摘要（提交截止日期为2012年10月1日）中填写项目的其余部分。连续第二年，研讨会将在研究会议之前的星期二（2月12日）增加一个教学部分“溶酶体101，溶酶体生物学和疾病基础”。这个4小时的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，为随后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，以便他们参加“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 9th annual WORLD Symposium will be held February 13-15, 2013 (Orlando, FL, USA). This research meeting is a multidisciplinary forum presenting the latest advances in basic science, translational research, and clinical trials for lysosomal diseases. The theme of the meeting will emphasize 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; 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 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 all lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Evidence of the synergistic nature of the WORLD Symposium has been outgrowth of the multi-center consortium "Lysosomal Disease Network" (U54 NS065768, a member of the Rare Diseases Clinical Research Network, RDCRN). 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 to share information that otherwise does not exist. The program is organized in six sessions extending from "basic research" through "translational research" to "clinical research". For each, the Program Committee will select two Invited Speakers and will fill the remainder of the program from submitted abstracts (submission deadline October 1, 2012). For the second straight year, the Symposium will add a didactic component "Lysosomes 101, Fundamentals of Lysosomal Biology and Disease" on the Tuesday (February 12) preceding the research meeting. Building on-but not supplanting-the curriculum of the previous year, this 4-hour educational session will offer attendees the basic concepts of biochemistry, immunology, molecular biology, and genetics of lysosomal disease, providing a common understanding for subsequent cutting edge research presentations. 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.