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

描述（由申请人提供）：此资助申请要求对题为“规划有效地将核酸输送到皮肤”的研讨会的部分支持。“一项1b期临床试验于2008年8月成功完成，其中针对导致先天性厚甲症（PC）的特定突变的siRNA通过注射给患者。该临床试验是基于2006年由PC项目和NIH/NIAMS/ORG赞助的科学会议上制定的计划。该临床试验在会议后两年内完成，在IPCC成立后不到四年的时间内完成。 迫切需要一个有效的、对病人友好的分娩计划。PC是一种理想的常染色体显性遗传性皮肤病模型，PC治疗和递送的进展将对皮肤病研究和治疗的整体进展产生积极影响。该会议由PC Project组织和共同赞助，PC Project是一家成立于2003年的非营利公共慈善机构。PC Project积极赞助国际PC联盟，该联盟由约50名国际合作者组成，致力于开发和提供治疗先天性甲肥厚的疗法。本赠款申请书所附的参考书目中引用了气专委各成员的出版物。 除了IPCC成员的进展报告外，2009年论坛还将通过不同领域专家的投入得到加强，包括对其他罕见皮肤病进行研究的科学家和临床医生。这次会议是必不可少的汇集这些专家谁一起将制定一项计划，以克服许多皮肤疾病，包括PC所面临的交付障碍。2009年IPCC科学会议的结果将是一个更有效的计划，更友好的病人和有效的皮肤交付。 会议将于2009年5月在皮肤病学研究学会年会之前举行。该网站将在传统中心在蒙特利尔，加拿大这是一个残疾人无障碍设施。会议还将有初级调查员参加，并将特别努力让妇女和少数民族参加讨论小组和发言。

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.