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

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