CIRM Center for Neuropsychiatric Stem Cell Proteomics
Research Objective This project will interrogate interactions, distribution, and function of high-confidence neuropsychiatric disorder risk proteins, and identify convergent pathobiology of patient genetic variants. Impact Datasets and stem cell resources…
Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models
Research Objective Our project will identify molecular and cellular changes induced by specific genetic variants implicated in schizophrenia and autism spectrum disorder in stem cells, neuroprogenitor cells and neurons. Impact…
hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders.…
Novel antisense therapy to treat genetic forms of neurodevelopmental disease.
Research Objective We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four…
AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder
Research Objective We propose a gene therapy for the treatment of a severe infantile epilepsy called CDKL5 Deficiency Disorder using CRISPR-mediated epigenetic editing Impact A transformative treatment for females affected…