This submission is a new application for the UCLA IDDRC, entitled UCLA Center for Translational Research in Neurodevelopment (UC-TRaN). The fundamental purpose of our Center is to provide an optimal environment for performing outstanding research into the causes, mechanisms, and treatments of intellectual and developmental disorders. The new leadership model in the center emphasizes collaborations among Center investigators, core personnel, and community partners, with support for research spanning basic research and clinical practice.
Our aims are 1) to create cutting edge and cost effective infrastructure resources in facilities and personnel for IDD investigators; 2) to provide integrated services within cores that support research from multidisciplinary teams and multiple levels of analysis; 3) to promote interactions between cores and among research investigators to promote collaborative, multidisciplinary and translational research, and 4) to create educational and outreach opportunities within the center, in collaboration with a West Coast Consortium of IDDRCs and community partners. We propose five interacting cores: 1) Administration, Education and Outreach; 2) Genetics, Genomics and BioInformatics, which performs genetic analysis, sequencing, expression, and provides Big Data resources; 3) Cells, Circuits and Systems analysis, supporting electrophysiology, human iPSC and cerebral organoid culture development, and optogenetics; 4) Structural and Functional Visualization, which provides access to and analysis of imaging tools from miniaturized microscopes through animal and human MRI; and 5) Clinical Translation, supporting clinical trial development, recruitment, phenotyping across species, and collecting biosamples. The Center proposes an exciting model research project, Neurophysiological biomarkers of cognition in Dup15 syndrome: From mouse models to patients. Dup15q11.2-13.1 is associated with marked cognitive impairment and other symptoms of developmental delay. The study examines a novel EEG biomarker in a cohort of children with Dup15q and in two separate mouse models of Dup15q syndrome, one defined by an overexpression of a single gene, UBE3A, and the other from a full duplication of the region. In the animal model the project will test three mechanism-based pharmacologic treatments: d-serine, rapamycin, and a GABA-A ?5 receptor subunit inverse agonist, to determine whether these treatments change cell activation patterns and behavior. Together, the project and cores support our primary goal of optimizing outcomes of individuals with intellectual and developmental disabilities by promoting outstanding translational research.
This is a new application for the UCLA IDDRC, the UCLA Center for Translational Research in Neurodevelopment (UC-TRaN). The Center is dedicated to improving the lives of individuals with intellectual and developmental disabilities by creating a research environment optimized for studying the basic cases and mechanisms leading to IDDs, and designing effective treatments and prevention strategies. Our center is strongly focused on building collaborations among researchers studying the same problems with different techniques, and partnering with the community to ensure scientific discoveries will benefit individuals. Our application builds on over 50 years of dedicated research into intellectual and developmental disorders.
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