UCLA Center for Translational Research in Neurodevelopment: UC-TRaN

NIH RePORTER · NIH · U54 · $1,298,372 · view on reporter.nih.gov ↗

Abstract

 DESCRIPTION (provided by applicant): 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 α5 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.

Key facts

NIH application ID
9836642
Project number
5U54HD087101-05
Recipient
UNIVERSITY OF CALIFORNIA LOS ANGELES
Principal Investigator
SUSAN Y BOOKHEIMER
Activity code
U54
Funding institute
NIH
Fiscal year
2020
Award amount
$1,298,372
Award type
5
Project period
2015-11-01 → 2020-07-31