MIND Institute Intellectual and Developmental Disabilities Research Center

描述（由申请人提供）：本申请寻求在加州大学建立一个智力和发育障碍研究中心（IDDRC）的资金，该中心将完全融入加州大学的现有资源并从中受益 戴维斯MIND研究所及其校园合作伙伴。MIND研究所是一个综合项目，在自闭症、脆性X综合征和其他IDD疾病的开创性研究方面得到国际认可。拟议的IDDRC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。该部分包括一项针对脆性X综合征儿童的临床试验，该试验将药物（洛伐他汀）与父母实施的干预相结合。新的结果措施侧重于语言和挑战行为将评估疗效。临床试验将伴随着专注于FMRI相关信号通路的研究，以阐明治疗实现其效果的机制，并确定指示治疗疗效和治疗反应个体差异的生物标志物。第二，我们建议纳入由NIH和其他联邦和私人来源资助的56个与缺碘症有关的项目。其中10个项目由国家排雷中心提供资金。这些项目涉及三个科学主题：缺碘症的综合生物行为特征、环境对缺碘症的影响和缺碘症的治疗。第三，我们提出了核心组成部分，以促进IDD的跨学科，转化研究。核心包括行政核心和临床翻译核心，后者侧重于人类参与者的招募和诊断特征。其余的核心将提供免疫功能，细胞和分子生物学和成像，以及环境污染物的测定（生物分析核心）;复杂人类行为的测量，特别是用于确定内在表型（神经行为分析核心）;用于评价突变啮齿动物模型和支持药物安全性和有效性临床前评价的啮齿动物行为测定（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).