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

IPCC 2009 研讨会：核酸有效输送至皮肤的规划

• 批准号: 7675916
• 负责人: Mary Schwartz
• 金额: $ 3.5万
• 依托单位: PACHYONYCHIA CONGENITA PROJECT
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-05-01 至 2009-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7675916
• 关键词: Applications Grants; Bibliography; Canada; Charities; Clinical Trials; Dermatology; Development; Disabled Persons; Injection of therapeutic agent; International; Minority; Modeling; Mutation; Nucleic Acids; Patients; Phase; Progress Reports; Publications; Request for Applications; Research; Research Personnel; Scientist; Site; Skin; Small Interfering RNA; Societies; Therapeutic; Woman; base; handicapping condition; meetings; member; methionylmethionine; skin disorder; symposium

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.

描述（由申请人提供）：本赠款申请要求对题为“有效递送核酸向皮肤的计划”的研讨会提供部分支持。 1B期临床试验在2008年8月成功完成，其中siRNA针对特定的突变，导致Pachyonychia congenita（PC）通过注射给患者进行。临床试验基于在2006年由PC Project和NIH/NIAMS/ORG赞助的科学会议上制定的计划。临床试验是在会议后的两年内完成的，距IPCC成立不到四年。 迫切需要一个有效，对患者友好型分娩的计划。 PC是一种理想的模型常染色体显性皮肤疾病，并且PC治疗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2003年成立的非营利性公共慈善机构PC Project组织和共同赞助。PC项目积极赞助国际PC财团，该集团由大约50个国际合作者组成，致力于开发和提供治疗剂，以治疗Pachyonychia congenita。 IPCC成员的出版物在此赠款申请书附带的参考书目中引用。 除了有关IPCC成员进展的报道外，2009年论坛还将通过包括科学家和临床医生在其他罕见皮肤疾病进行研究（包括科学家和临床医生）的专家的意见来加强。这次会议对于将那些将制定计划的专家组合在一起，以克服包括PC在内的许多皮肤疾病所面临的递送障碍的计划至关重要。 2009年IPCC科学会议的结果是，将有效的患者友好和有效地分娩给皮肤的有效计划。 会议将于2009年5月在调查性皮肤病学会年会之前举行。该地点将在加拿大蒙特利尔的常规中心，该中心是一个残障的可访问设施。会议还将涉及初级调查人员，并将特别努力将妇女和少数民族包括在讨论小组和演讲中。