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ZENODO
Dataset . 2024
License: CC BY
Data sources: ZENODO
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Single-cell and TCR sequencing analysis of peripheral blood mononuclear cells from Duchenne muscular dystrophy patients before and after dosing with AAV9 mini-dystrophin

Authors: Emami, Michael; Brimble, Mark; Espinoza, Alejandro; Owens, Jane; Whiteley, Laurence; Casinghino, Sandra; Lanz, Thomas; +8 Authors

Single-cell and TCR sequencing analysis of peripheral blood mononuclear cells from Duchenne muscular dystrophy patients before and after dosing with AAV9 mini-dystrophin

Abstract

We used single-cell RNA sequencing and T cell receptor (TCR) sequencing on peripheral blood mononuclear cells from five patients with Duchenne muscular dystrophy before and after dosing with PF-06939926 fordadistrogene movaparvovec. Included in this repository are the following: SS_MS_DMD_AAV_integrated.rds is a Seurat object containing single-cell gene expression for pre and post time points from all subjects. TCR information is included in the metadata slot. Supplemental Data File 1 PostvsPre_DEG_tables_ANNOTATED by_cell_type.xlsx contains the differentially expressed genes between the pre and post time points by immune cell population. MS_DMD_all_conga_final.h5ad is an AnnData object containing the final output of the CoNGA analysis. Results of the analysis are stored in adata.uns['conga_results']. See the github page for more information on the data structure: CoNGA github

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