Obstacles to Translating Basic Knowledge of Genetic Skin Disease into Therapies

将遗传性皮肤病的基本知识转化为治疗方法的障碍

• 批准号: 7294523
• 负责人: Ervin HAROLD Epstein
• 金额: $ 2.5万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-09-30 至 2006-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7294523
• 关键词: genetic disorder; meeting /conference /symposium; skin disorder; skin disorder chemotherapy; travel

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.

描述（由申请人提供）： 本申请请求支持召开一次会议，解决阻碍将遗传性皮肤病基因缺陷的基础知识转化为这些疾病的实用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中的障碍以及对其使用的免疫障碍；监管障碍；以及药物发现和开发的财务障碍。 会议首先将描述这些障碍，其次将重点讨论克服这些挑战的战略。长期目标是促进对那些目前已知其根本原因的皮肤病的转化研究，预计该研究的经验教训最终将不仅适用于更广泛的皮肤病，目前尚不清楚根本缺陷，但更普遍的是分子靶向疗法的开发。