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.
|Jordan, Maria C; Zheng, Yi; Ryazantsev, Sergey et al. (2005) Cardiac manifestations in the mouse model of mucopolysaccharidosis I. Mol Genet Metab 86:233-43|