Strategies for Therapy of MPS and Related Diseases

描述（由申请人提供）： 此应用程序是支持一个会议上“的治疗策略， MPS和相关疾病”将在加州洛杉矶大学举行 洛杉矶，2001年6月21日至24日。MPS（粘多糖沉积症）是可遗传的 溶酶体功能疾病是慢性的、进行性的，涉及许多 器官，并导致各种残疾以及过早死亡。最近 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， 储藏疾病，包括酶替代疗法的临床试验。在 与此同时，改变这些疾病的过程中存在许多困难， 其中，最大的困难，就是改变。 疾病的神经系统部分。会议的目标是a） 传播关于发病机制的新发现，包括新发现的基因， 巨噬细胞源性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，如如何穿过血脑 屏障，酶靶向，基因治疗载体优化，免疫 反应、底物剥夺和干细胞治疗。我们将邀请25-30 研究人员在该领域的前沿是核心发言人， 预计共有约一百名专业人士参加。不是所有的演讲者 空缺已经填补，以便从下列国家中挑选更多的发言者： 在新的科学发展的基础上也进行了摘要。本次会议 将与全国MPS协会年会同时举行 股份有限公司、预计将有200个家庭参加。科学 会议将开放给家庭，反之亦然，并将有一个 两人之间进行正式和非正式互动的机会数目 组这次会议的最终目的是促进和加快 MPS和相关溶酶体贮积症的有效治疗研究 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.