MIND Institute Intellectual and Developmental Disabilities Research Center

描述(由申请人提供)：本申请寻求资助在加州大学建立智力和发育障碍研究中心(IDDRC)，该中心将完全整合到加州大学的现有资源中并受益于该中心 戴维斯心智研究所及其校园合作伙伴。心智研究所是一个综合性项目，在自闭症、脆性X综合征和其他IDD条件的开创性研究方面得到国际认可。拟议的国际发展和发展中心包括三个组成部分。首先，我们提出了一个研究组成部分，解决了多模式治疗方法的重点领域。这一组成部分包括一项针对脆性X综合征儿童的临床试验，该试验结合了一种药物(洛伐他汀)和父母实施的干预措施。关注语言和挑战行为的新结果衡量标准将评估疗效。临床试验将伴随着侧重于功能磁共振成像相关信号通路的研究，以阐明治疗取得效果的机制，并确定指示治疗效果和治疗个体差异的生物标记物。其次，我们建议纳入由美国国立卫生研究院和其他联邦和私人来源资助的56个与缺碘相关的项目。其中10个项目由NICHD提供资金。这些项目涉及三个科学主题：IDD的综合生物行为特征、对IDD的环境贡献以及IDD的治疗。第三，我们提出了促进IDD跨学科、转化性研究的核心组成部分。核心包括行政核心和临床翻译核心，后者侧重于招募和诊断参与者的特征。其余核心将提供免疫功能、细胞和分子生物学和成像以及环境污染物的分析(生物分析核心)；复杂人类行为的测量，特别是用于建立内部表型的测量(神经行为分析核心)；用于评估突变啮齿动物模型和支持药物安全性和有效性的临床前评估的啮齿动物行为分析(啮齿动物行为核心)；以及来自人类和动物研究的复杂多维数据的管理和分析(生物统计学、生物信息学和研究设计)。

DESCRIPTION (provided by applicant): The present application seeks funding to establish an Intellectual and Developmental Disabilities Research Center (IDDRC) at the University of California, which will be fully integrated into, and benefit from, the existing resources of the UC Davis MIND Institute and its campus partners. The MIND Institute is an integrated program that is internationally recognized for ground-breaking studies of autism, fragile X syndrome, and other IDD conditions. The proposed IDDRC includes three components. First, we propose a research component that addresses the focus area of Multi-Modal Treatment Approaches. This component includes a clinical trial targeting children with fragile X syndrome that combines a drug (lovastatin) with a parent-implemented intervention. Novel outcome measures focusing on language and challenging behavior will evaluate efficacy. The clinical trial will be accompanied by studies focused on FMRI-related signaling pathways so as to illuminate the mechanisms by which treatment achieves its effects and to identify biomarkers that indicate treatment efficacy and individual differences in response to treatment. Second, we propose for inclusion 56 IDD-related projects funded by NIH and other federal and private sources. Ten of the projects are funded by NICHD. These projects address three scientific themes: Integrated Biobehavioral Characterization of IDD, Environmental Contributions to IDD, and Treatments for IDD. Third, we propose core components to facilitate interdisciplinary, translational research in IDD. The cores include an Administrative Core and a Clinical Translational Core, with the latter focused on recruitment and diagnostic characterization of human participants. The remaining cores will provide assays of immune function, cellular and molecular biology and imaging, and environmental contaminants (Biological Analysis Core); measures of complex human behavior, particularly for establishing endophenotypes (Neurobehavioral Analysis Core); assays of rodent behavior for evaluating mutant rodent models and supporting preclinical evaluations of drug safety and efficacy (Rodent Behavior Core); and the management and analysis of complex multidimensional data derived from human and animal studies (Biostatistics, Bioinformatics, and Research Design).