NIH Support of Conferences and Scientific Meetings

描述(申请人提供)：唐氏综合征(DS)的特征是特定的学习/记忆(L/M)缺陷和脑异常。这些特征中的许多都是在出生后发展起来的，这表明有可能通过有效的产后治疗来防止它们的发病或减轻它们的严重程度。这一目标得到了最近通过几种药物治疗在修复Ts65Dn DS小鼠模型中L/M缺陷方面的成功支持。将这些观察结果扩展到安全、有效的人体临床试验需要吸收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有效性，并探索广泛的药物治疗方案，以确定哪些是最具潜力的。没有集中于DS智能障碍的基础、临床和翻译研究的会议 已经举办了大约5年。这项申请要求提供资金，以支付召开会议的费用，以便将来自这些不同研究领域的研究人员聚集在一起。参与者将包括公认的DS和非DS研究人员，他们的研究直接或间接地告知DS认知表型的各个方面和/或这些特征对药物治疗的反应。会议将于2013年4月在华盛顿特区举行，为期三天。预计这次会议将导致关于药物治疗DS认知缺陷的新假说，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.