Year: 2020
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Leveraging novel AAV capsids and CRISPR/Cas9 gene editing as a gene therapeutic approach to target the XDP-SVA
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SVA-mediated chromatin re-organisation in XDP
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Characterization of SVA retrotransposon dynamics in iPS-derived organoid models and postmortem tissue of X-Linked Dystonia-Parkinsonism
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Rapid drug discovery of TAF1 deficiency therapeutics in vivo
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Presence and Role of DNA and RNA G- and C-quadruplex Structures in the Pathogenesis of XDP
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Identification of pathogenic mechanisms underlying neurodegeneration of XDP neurons generated by direct conversion of fibroblasts
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Mechanistic insights into the role of a proposed novel genetic modifier of X-linked Dystonia-Parkinsonism
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Dissecting the influence of the XDP-linked SVA insertion in the TAF1 locus on promoter-proximal pausing
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Deciphering the role of histone cirtullination and inflammation in XDP
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Developing a cell-based system to screen compounds countering XDP-SVA mediated transcriptional repression