NIH Support of Conferences and Scientific Meetings

描述（由申请人提供）：唐氏综合征（DS）的特征是特定的学习/记忆（L/M）缺陷和大脑异常。许多这些特征在出生后发展，这表明有效的产后治疗方法可以预防其发作或降低其严重程度。这一目标得到了最近成功的支持，与几种药物治疗，在拯救L/M赤字的Ts 65 Dn小鼠模型的DS。将这些观察结果扩展到安全、有效的人体临床试验，需要同化21号染色体基因上的功能信息，详细了解DS认知表型，在人体试验之前使用适当的模型系统来测试药物的疗效，并探索广泛的药理学方案，以确定最具潜力的方案。没有一次会议关注DS智力残疾的基础、临床和转化研究 已经举行了大约5年。这项申请要求提供资金，以支付会议的费用，以汇集来自这些不同研究领域的研究人员。参与者将包括已建立的DS和非DS研究者，其研究直接或间接地告知DS认知表型和/或这些特征对药物治疗的反应。会议将于2013年4月在华盛顿举行，为期三天。预计这次会议将导致关于药物治疗DS认知缺陷的新假设，促进新的合作，并将具有新专业知识的新研究人员带入DS领域。

DESCRIPTION (provided by applicant): Down syndrome (DS) is characterized by specific learning/memory (L/M) deficits and brain abnormalities. Many of these features develop after birth, suggesting the possibility of effective postnatal therapeutics to prevent their onset or reduce their severity. This goal is supported by recent successes, with several pharmacological treatments, in rescuing L/M deficits in the Ts65Dn mouse model of DS. Extending these observations to safe, effective human clinical trials requires assimilation of functional informatin on chromosome 21genes, detailed understanding of the DS cognitive phenotype, use of appropriate model systems to test efficacy of drugs prior to human trials, and exploration of a broad range of pharmacological regimens in order to identify those of the greatest potential. No conference focusing on basic, clinical and translational research for intellectual disability in DS has been held in approximately 5 years. This application requests funding to cover expenses for a conference to bring together investigators from these diverse research areas. Participants will include established DS and non-DS investigators whose research directly or indirectly informs aspects of the DS cognitive phenotype and/or responses of these features to pharmacological therapy. The meeting will be held for three days in Washington DC in April 2013. It is anticipated that this conference will lead to new hypotheses regarding pharmacological treatment of cognitive deficits in DS, foster new collaborations and bring new researchers with novel expertise into the field of DS.