2015 Lysosomal Disease Gordon Research Conference and Gordon Research Seminar

 描述（由申请人提供）：溶酶体疾病GRC于2011年在德克萨斯州加尔维斯顿的Galvez酒店举行了第一次会议，并于2013年在意大利卢卡（巴尔加）的Renaissance托斯卡纳Il Ciocco度假村举行了第二次会议。我们是由GRC组织的170多个年度会议中最新的一个，GRC是一个以其高质量，前沿学术会议而闻名的组织，并且确实是唯一一个在2013年审查中被批准为重复会议的新GRC。溶酶体疾病代表了一组由广泛蛋白质的遗传缺陷引起的50多种疾病，其中许多仍然知之甚少。这些疾病影响许多器官系统，最明显的是大脑，导致慢性疾病和受影响个体的死亡。2015年溶酶体疾病GRC将再次为解决涉及溶酶体疾病的重大挑战提供一个关键场所，包括需要更好地理解（1）更大的溶酶体系统在维持健康和疾病中的细胞稳态中的作用，（2）神经元和大脑功能障碍的溶酶体疾病致病机制，以及其他细胞类型/器官/系统，（3）溶酶体和非溶酶体蛋白的特定功能，其中的缺陷引起溶酶体疾病，（4）溶酶体功能障碍和其它慢性神经变性病症（例如，阿尔茨海默氏症和帕金森氏症），以及（5）常染色体和X连锁溶酶体疾病中杂合性可导致细胞和临床疾病的程度。此外，通过严格的临床前研究来开发针对这些疾病的新的和创新的疗法以及（7）启动和完成有效的临床试验也是一项挑战。为了解决这些重要的主题，我们迄今为止邀请了27位在溶酶体疾病领域工作的领先的国际知名科学家和临床医生作为演讲者和讨论者。每个人都接受了我们的邀请，并将加入我们预计将增加的170名与会者;我们预计其中整整三分之一将是初级调查员， 处于职业生涯早期阶段的博士后研究员和研究生。重要的是，我们2011年会议的成功导致GRC组织选择溶酶体疾病会议作为戈登研究研讨会（GRS）的主办方，这是一个由研究生和早期博士后研究员组织和运行的特别会议。我们的2015年会议再次被选为主办GRS。GRS的存在确保了下一代溶酶体疾病研究人员中最优秀和最聪明的人也将成为GRC的一部分。作为GRC和GRS会议的一部分，我们相信讨论和交叉施肥的想法和方法不仅会加速我们对溶酶体系统在健康和疾病中的作用的理解，而且会使我们更接近许多这些疾病的有效疗法。

 DESCRIPTION (provided by applicant): The Lysosomal Disease GRC had its first meeting at the Hotel Galvez in Galveston, Texas in 2011, and its second at the Renaissance Tuscany Il Ciocco Resort in Lucca (Barga), Italy in 2013. We are one of the newest of over 170 annual conferences organized by the GRC, an organization known world-wide for its high-quality, cutting-edge academic conferences, and indeed are the only new GRC approved as a repeat conference in their 2013 review. Lysosomal Disease represents a group of more than 50 disorders caused by inherited defects in a wide spectrum of proteins, many of which remain poorly understood. These disorders affect many organ systems, most notably brain, leading to chronic illness and death of affected individuals. The 2015 Lysosomal Disease GRC will again offer a critical venue for addressing major challenges involving lysosomal diseases, including the need for better understanding of (1) the role of the greater lysosomal system in maintaining cell homeostasis in health and disease, (2) lysosomal disease pathogenic mechanisms underlying the dysfunction of neurons and brain, as well as other cell types/organs/systems, (3) specific functions of lysosomal and non-lysosomal proteins, defects in which cause lysosomal disease, (4) the relationship between lysosomal dysfunction and other chronic neurodegenerative conditions (e.g., Alzheimer's and Parkinson's), and (5) the extent to which heterozygosity in autosomal and X-linked lysosomal diseases can lead to cellular and clinical disease. Additionally there is the challenge (6) through rigorous preclinical studies to develop new and innovative therapies for these disorders coupled with (7) initiating and completing effective clinical trials. To address these important topics we have to date invited, as speakers and discussants, 27 leading and internationally known scientists and clinicians working in the field of lysosomal disease. Each has accepted our invitation and will join what we anticipate to be an additional 170 attendees; fully one-third of whom we anticipate will be junior investigators, postdoctoral fellows and graduate students who are at the early stages of their careers. Importantly, the success of our 2011 meeting led to the GRC Organization selecting the Lysosomal Disease meeting as a host for a Gordon Research Seminar (GRS), a special meeting organized and run by graduate students and early postdoctoral fellows. Our 2015 meeting has again been selected to host a GRS. The presence of the GRS insures that the very best and the brightest of the next generation of lysosomal disease researchers will be part of the GRC as well. The discussion and cross fertilization of ideas and approaches occurring as part of the GRC and GRS meetings we believe will accelerate not only our understanding of the role of the lysosomal system in health and in disease, but also move us closer to effective therapies for many of these disorders.