Strategies for Therapy of MPS and Related Diseases

描述(由申请人提供)： 本申请书是为了支持一个名为“治疗的策略”的会议。 MPS及相关疾病研讨会将在加州大学洛杉矶分校举行 2001年6月21日至24日在洛杉矶举行。粘多糖病(MPS)是可遗传的 溶酶体功能疾病是慢性的、进行性的，涉及许多 并导致各种残疾和过早死亡。近期 克隆基因、重组酶和动物模型的可用性 在这些与溶酶体密切相关的研究中产生了一系列的研究活动 储存疾病，包括酶替代疗法的临床试验。在… 与此同时，改变这些疾病的进程有许多困难 变得显而易见，其中最令人烦恼的是改变 这种疾病的神经学成分。会议的目标是) 传播关于发病机制的新发现，包括新发现的基因和 巨噬细胞衍生细胞在神经元疾病中的作用，以及b)讨论 治疗领域的主要问题，如跨越血脑的方法 屏障、酶靶向、基因治疗载体的优化、免疫 反应、底物剥夺和干细胞治疗。我们将邀请25-30人 该领域最前沿的调查人员将成为核心演讲者，以及 预计总共将有大约100名专业人士参加。并非所有演讲者 空位已填满，以允许从以下位置选择额外的发言者 在新的科学发展的基础上也进行了摘要。这次会议 将与全国MPS协会年会同时举行 Inc.，预计将有大约200个家庭参加。科学的 会议将向家属开放，反之亦然，并将有 两者互动的正式和非正式机会的数量 组。本次会议的最终目标是促进和加快 多发性硬化症的有效治疗及相关溶酶体储存的研究 疾病。

DESCRIPTION (provided by applicant): This application is for the support of a meeting on "Strategies for Therapy of MPS and Related Diseases" to be held at the University of California Los Angeles on June 21-24, 2001. The MPS (mucopolysaccharidoses) are heritable diseases of lysosomal function that are chronic, progressive, involve numerous organs and cause a variety of disabilities as well as early death. Recent availability of cloned genes, recombinant enzymes and animal models has generated a burst of research activity in these and closely related lysosomal storage diseases, including clinical trials of enzyme replacement therapy. At the same time, many difficulties in altering the course of these disorders have become apparent, the most vexing of which is the difficulty of altering the neurologic component of the disease. The goals of the meeting are to a) disseminate new findings on pathogenesis, including newly identified genes and the role of macrophage-derived cells in neuronal disease, and b) to discuss major issues in the area of therapy, such as ways to cross the blood-brain barrier, enzyme targeting, optimization of vectors for gene therapy, immune reactions, substrate deprivation, and stem cell therapy. We will invite 25-30 investigators at the cutting edge of the field to be the core speakers, and expect a total of about a hundred professional attendees. Not all the speaker slots have been filled, to permit selection of additional speakers from abstracts as well on the basis of new scientific developments. This meeting will be held in conjunction with the annual meeting of the National MPS Society Inc., which expects an attendance of about 200 families. The scientific meetings will be open to the families and vice-versa, and there will be a number of formal and informal opportunities for interaction between the two groups. The ultimate goal of this meeting is to facilitate and accelerate research for effective therapy of the MPS and related lysosomal storage diseases.