2015 Lysosomal Disease Gordon Research Conference and Gordon Research Seminar

 描述(申请人提供)：溶菌体疾病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(Barga)的文艺复兴托斯卡纳Il Ciocco度假村举行了第二次会议。我们是GRC组织的170多个年度会议中最新的一个，GRC是一个以高质量、尖端的学术会议而闻名世界的组织，而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50多种疾病，这些疾病是由广泛蛋白质谱中的遗传缺陷引起的，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，导致慢性病和受影响个人的死亡。2015年溶酶体疾病GRC将再次为解决涉及溶酶体疾病的主要挑战提供一个关键场所，包括需要更好地了解(1)更大的溶酶体系统在维持健康和疾病中细胞动态平衡方面的作用，(2)溶酶体疾病导致神经元和大脑功能障碍的致病机制，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，导致溶酶体疾病的缺陷，(4)溶酶体功能障碍与其他慢性神经退行性疾病(例如阿尔茨海默氏症和帕金森氏症)之间的关系，以及(5)常染色体和X连锁溶酶体疾病的杂合性可导致细胞性疾病和临床疾病的程度。此外，通过严格的临床前研究为这些疾病开发新的和创新的疗法，以及启动和完成有效的临床试验，这是一个挑战。为了解决这些重要的主题，我们迄今邀请了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人员， 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，2011年会议的成功促使GRC组织选择溶虫体疾病会议作为戈登研究研讨会(GRS)的东道主，这是一个由研究生和博士后研究员组织和运营的特别会议。我们2015年的会议再次被选为GRS的东道主。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，想法和方法的讨论和交叉融合不仅将促进我们对溶酶体系统在健康和疾病中的作用的理解，而且还将使我们更接近于对许多此类疾病的有效治疗。

 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.