Obstacles to Translating Basic Knowledge of Genetic Skin Disease into Therapies

描述（由申请人提供）： 该申请要求支持召开一次会议，讨论阻碍将遗传性皮肤病基因缺陷的基本知识转化为这些疾病的实际分子靶向疗法的障碍。要解决的障碍将包括识别和生产有用分子的障碍，将分子输送到活体皮肤的障碍和使用它们的免疫障碍;监管障碍;以及药物发现和开发的财务障碍。 会议将首先阐明这些障碍，然后着重讨论克服这些障碍所带来的挑战的战略。长期目标是促进对那些根本原因现在已知的皮肤病的转化研究，并且预计该研究的教训最终不仅适用于更广泛的皮肤病，其根本缺陷现在还不知道，而且更普遍地适用于分子靶向疗法的开发。

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.