Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons
Approximately 5,600 people in the U.S. are diagnosed with ALS each year. The incidence of ALS is two per 100,000 people, and it is estimated that as many as 30,000…
Modeling the genetic basis of psychopathology in schizophrenia and autism
Research Objective We propose to apply scalable platforms for the characterization of CNVs and genes coupled with detailed characterizations of neurodevelopment of the 4 major CNV loci in model systems…
Multi-gene modulation to rescue CNS-associated microdeletion syndromes
Research Objective Chromosomal microdeletion syndromes result in severe neuropsychiatric syndromes and lack therapy. This proposal will define critical genomic regions needed to generate new tools for functional rescue. Impact This…
Defining Neurovascular Metabolism in Neurodevelopmental and Neuropsychiatric Disorders
Research Objective Neuropsychiatric disorders correlate to impaired metabolism, though understudied. We will describe how metabolism impacts disorders etiology and identify new, tractable therapeutic strategies. Impact We will uncover metabolic drivers…
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