Tenth International Congress on Ceroid Lipofuscinoses

第十届国际蜡样质脂褐质病大会

• 批准号: 6941069
• 负责人: Glyn Dawson
• 金额: $ 3万
• 依托单位: UNIVERSITY OF CHICAGO
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-03-01 至 2006-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6941069
• 关键词: endopeptidases; esterase; gene therapy; meeting /conference /symposium; nervous system disorder chemotherapy; neuronal ceroid lipofuscinosis; protein degradation; stem cell transplantation; travel

DESCRIPTION (provided by applicant): Conference support is sought for the Tenth International Congress on Ceroid Lipofuscinoses (Battens disease) to be held at the Hanasaari Congress Center, Helsinki, Finland from June 8th to 12th, 2005. We anticipate 130 participants from all over the world and support is requested for some of the US participants. Matching funds are being solicited from the EU. The Congress center has capacity to accommodate 92 persons and the rest of the participants can be accommodated at nearby hotels. It makes an ideal congress venue due to the fact that the participants are gathered together but on the other hand it is readily accessible from Helsinki. The Conference center is fully equipped for handicapped access. This is essential both for the participation of scientists and for the ability of parents and their wheelchair bound children to attend and participate in the meeting. The Battens group of childhood neurodegenerative disorders involve at least 10 different genes and collectively are the most common inherited neurodegenerative diseases of children. They are characterized by vision loss, seizures and mental retardation. All are believed to involve impaired lysosomal protein degradation although only two of the genes involved, palmitoyl;protein thioesterase and tripeptidylpeptidase, have been identified as enzymes. The proteins coded by CLN3, 5,6 and 8 are of unknown function and one focus of this meeting will be to use proteomics, in combination with biochemistry and cell biology, to try to determine this function. Other potentially exciting areas to be covered will include the use of cord blood-derived stem cells, gene therapy, nutritional therapy, drug therapy and the use of transgenic animal models to develop effective treatments. The previous 9 Congresses have brought together physicians, basic researchers and parents and have provided a forum for young researchers, especially females, to present their work orally and participate in lively discussions. Helsinki was the site of the 6th Congress and the practice has been to alternate the Conference between Europe and the USA, resulting in the establishment of international collaborative research and facilitating better understanding of this devastating disease.

说明（由申请人提供）： 第十届国际蜡样脂褐质沉积症（巴滕斯病）大会将于 2005 年 6 月 8 日至 12 日在芬兰赫尔辛基 Hanasaari 会议中心举行。我们预计将有来自世界各地的 130 名与会者世界各地，并要求一些美国参与者提供支持。正在向欧盟征求配套资金。会议中心可容纳 92 人，其余与会者可入住附近的酒店。由于与会者聚集在一起，因此它成为理想的会议场所，但另一方面，从赫尔辛基可以轻松到达。会议中心设施齐全，适合残疾人士使用。这对于科学家的参与以及家长及其坐轮椅的孩子出席和参与会议的能力至关重要。 Battens 组儿童神经退行性疾病涉及至少 10 种不同的基因，总体上是儿童最常见的遗传性神经退行性疾病。他们的特点是视力丧失、癫痫发作和智力低下。所有这些都被认为涉及受损的溶酶体蛋白质降解，尽管只有其中两个相关基因，即棕榈酰蛋白硫酯酶和三肽基肽酶，已被鉴定为酶。 CLN3、5,6和8编码的蛋白质功能未知，本次会议的一个焦点将是利用蛋白质组学，结合生物化学和细胞生物学，尝试确定其功能。其他可能令人兴奋的领域将包括使用脐带血干细胞、基因治疗、营养治疗、药物治疗以及使用转基因动物模型来开发有效的治疗方法。前九届大会汇聚了医生、基础研究人员和家长，为年轻研究人员，特别是女性研究人员提供了一个口头展示其工作并参与热烈讨论的论坛。赫尔辛基是第六届大会的举办地，惯例是在欧洲和美国轮流举行会议，从而建立了国际合作研究并促进更好地了解这种毁灭性疾病。