Translational Opportunities for the Heritable Disorders of Connective Tissue

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DESCRIPTION (provided by applicant): The proposed meeting on "Translational Opportunities for the Heritable Disorders of Connective Tissue" is scheduled for July 10-14, 2011, in Portland, OR. This meeting will fill a critical need, since the last meeting on heritable disorders of connective tissue was held more than a decade ago. A summary of the results and recommendations from the last meeting in 2000 was published (Sakai, LY, Byers, PH, and Ramirez, F. Matrix Biology 21:7-13, 2002 PMID:11827787). As in the previous meeting, the proposed meeting will include presentations that span the full range of topics in this area-from the identification of new disease genes to the unique connective tissue pathways that are involved in disease pathogenesis. However, due to the tremendous progress in the last decade, the proposed meeting will focus on prospects for new therapies for heritable disorders of connective tissue. The specific aims of the meeting are (1) to share results, discuss, and critically assess novel therapies; (2) to identify novel molecular or cellular mechanisms as promising targets for future therapies; (3) to define current opportunities for translating knowledge of basic mechanisms into new therapies. In addition, the meeting will stimulate new collaborations between investigators and will promote the research interests of young scientists. As in the past, a report of the meeting proceedings and recommendations will be prepared and published by the meeting organizers. The meeting will precede the Annual Meeting of the National Marfan Foundation, which will also be held in Portland, OR. The Marfan Syndrome was the first genetic disorder to be described by McKusick as a "heritable disorder of connective tissue." Today the National Marfan Foundation continues to play a lead role in the Coalition of Heritable Disorders of Connective Tissue. Others in the Coalition include the Osteogenesis Imperfecta Foundation, Pseudoxanthoma Elasticum (PXE) International, Dystrophic Epidermolysis Bullosa Research Association of America (DebRA), the Ehlers-Danlos National Foundation, the Little People of America, and the Loeys-Dietz Syndrome Foundation. Research relevant to each of these disorders represented in the Coalition will be presented; progress will be assessed and recommendations for future directions will be made. The overall goal of the meeting will be to stimulate the translation of novel basic information into new therapies for the heritable disorders of connective tissue. We anticipate that discussions at the meeting as well as our summary of the proceedings and recommendations for future directions will help to guide research in this area and will contribute to more rapid advances in translational medicine for the heritable disorders of connective tissue. PUBLIC HEALTH RELEVANCE: This application is to support a special meeting on "Translational Opportunities for the Heritable Disorders of Connective Tissue." The goals of the meeting are to critically assess recent progress in developing novel therapies for these unique disorders, to identify promising targets for future therapies, to stimulate new collaborations, and to promote areas in which rapid advances can be made. A summary of progress and recommendations will be published.