Figure 1.

Experimental design of scRNAseq analyses performed in Fanconi anemia group A patients 2-5 years afer lentiviral-mediated gene therapy. Four Fanconi anemia group A (FA-A) patients who had been treated with ex vivo lentiviral-mediated gene therapy in the absence of conditioning were included in this study. At 2-5 years post gene therapy, these patients harbored a chimeric population of corrected and uncorrected hematopoietic stem and progenitor cells (HSPC) in their bone marrow (BM). Aliquots of BM CD34⁺ cells from these patients were sorted and processed for single-cell RNA-seq. Bioinformatic analyses comparatively investigated changes in the transcriptional program of corrected versus uncorrected HSPC, co-existing in each of the gene therapy-treated patients. HSC: hematopoietic stem cell.