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

  • Alejandro Espinoza (Contributor)
  • Elizabeth M McNally (Contributor)
  • Jane Owens (Contributor)
  • S. Armando Armando Villalta (Contributor)
  • Mark Allen Brimble (Contributor)
  • Paul G. Thomas (Contributor)
  • Matteo Pellegrini (Contributor)
  • Thomas A. Lanz (Contributor)
  • Philip K. Farahat (Contributor)
  • Laurence O. Whiteley (Contributor)
  • Michael R. Emami (Contributor)
  • Sandra R. Casinghino (Contributor)
  • Melissa J. Spencer (Contributor)
  • Stefan A. Schattgen (Contributor)
  • Courtney S. Young (Contributor)

Dataset

Description

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
Date made availableOct 15 2024
PublisherZENODO

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