IPCC 2009 Symposium: Planning for Effective Delivery of Nucleic Acids to Skin

描述（由申请人提供）：本拨款申请要求部分支持题为“规划核酸有效输送到皮肤”的研讨会。2008年8月，一项1b期临床试验成功完成，该试验将siRNA靶向一种导致先天性厚甲癣（Pachyonychia Congenita， PC）的特定突变，通过注射给患者。临床试验是根据PC项目和NIH/NIAMS/ORG赞助的2006年科学会议制定的计划进行的。临床试验是在会议之后的两年内完成的，从IPCC成立到现在不到四年。

DESCRIPTION (provided by applicant): This grant application requests partial support for a symposium entitled "Planning for Effective Delivery of Nucleic Acids to Skin." A Phase 1b clinical trial was successfully completed in August 2008 in which siRNA targeted to a specific mutation causing Pachyonychia Congenita (PC) was delivered by injection to a patient. The clinical trial was based on the plan developed at the 2006 scientific meeting sponsored by PC Project and NIH/NIAMS/ORG. The clinical trial was achieved within two years following the meeting and in less than four years from the formation of the IPCC. A plan for effective, patient-friendly delivery is urgently needed. PC is an ideal model autosomal dominant skin disorder and progress in PC therapy and delivery will have a positive impact on the overall progress of skin disease research and treatment. The meeting is organized and co-sponsored by PC Project, a nonprofit public charity, established in 2003. PC Project actively sponsors the International PC Consortium, a group of approximately 50 international collaborators dedicated to the development and delivery of therapeutics to treat Pachyonychia Congenita. Publications by various IPCC members are cited in the bibliography attached to this grant application. In addition to reports of progress from IPCC members, the 2009 forum will be strengthened by input from experts in diverse fields including scientists and clinicians conducting studies for other rare skin disorders. This meeting is essential to bring together those experts who together will formulate a plan to overcome the delivery barrier faced by many skin disorders including PC. An effective plan for more patient-friendly and effective delivery to skin will be the result of the 2009 IPCC scientific meeting. The meeting will be held May 2009 just prior to the Annual Meeting of the Society of Investigative Dermatology. The site will be at the Conventional Center in Montreal, Canada which is a handicapped accessible facility. The meeting will also involve junior investigators and a special effort will be made to include women and minorities in the discussion groups and presentations.