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An In Vivo, iPSC-Based, Model of Autism

State/Province Full: 
United States

This Trailblazer study focuses on the identification of specific functional and morphological neuronal abnormalities that may underlie autism. Although the proof-of-principle experiments start with Fragile X patients, as these provide the best cell lines to validate the approach proposed, it is clear that the logical next step extension of these studies is into autism of other etiologies. Their novel strategy is to obtain fibroblasts from specific classes of very-well-characterized Fragile X patients, derive induced pluripotent stem cells (iPSCs) from the fibroblasts, differentiate neural cells from the iPSCs, and co-transplant paired cell populations, labeled with different fluorescent proteins, into the brains of mice for detailed in vivo analysis of morphology and physiology. This technique, which has never been done before, will allow scientists for the first time to view active ASD neurons, thus clarifying the nature of ASD physiology and help identify molecular targets for therapeutics.