Obstacles to Translating Basic Knowledge of Genetic Skin Disease into Therapies

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DESCRIPTION (provided by applicant): This application requests support for a Conference addressing the Obstacles impeding translation of fundamental knowledge of gene defects in heritable skin disorders into practical, molecularly-targeted therapies for these diseases. The Obstacles to be addressed will include Obstacles to Identification and Production of Useful Molecules, Obstacles to the Delivery of Molecules into the Living Skin and immunologic Obstacles to their Use; Regulatory Obstacles; and Financial Obstacles to Drug Discovery and Development. The Conference first will delineate these obstacles and second will focus on strategies for overcoming the challenges they present. The long-term goal is to stimulate translational research for those skin diseases in which the fundamental cause now is known, and it is expected that the lessons of that research also eventually will be applicable not only to the broader range of skin diseases for which the fundamental defect now is not known but also more generally to the development of molecularly-targeted therapies.