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Patient-derived brain organoids uncover reversible cellular abnormalities in Pitt-Hopkins Syndrome

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NIAID Data Ecosystem2026-05-02 收录
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https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE159392
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We evaluate cellular phenotypic abnormalities and alterations in molecular pathways due to mutations in the gene TCF4 in cells derived from Pitt-Hopkins patients, in comparison with respective controls (parents). Comparison between RNA-Seq libraries of neural progenitor cells and neurons derived from Pitt-Hopkins Syndrome patients and respective parents of matching sex. Libraries for each individual include three biological replicates.
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2025-02-25
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