GEOapplication/xmlftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE292nnn/GSE292605/primaryOK200TranscriptomicsHomo sapiensExpression profiling by high throughput sequencinghttps://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE292605GEOGSEfalseClonal analysis and transcriptional profiling of ex vivo expanded mobilized peripheral blood hematopoietic stem and progenitor [bulkRNAseq]Clonal analysis and transcriptional profiling of ex vivo expanded mobilized peripheral blood hematopoietic stem and progenitor. We characterized mPB CD34+ drug products and their ex-vivo expanded samples (4 and 7 days) cell populations (n=2 patients from 10.1056/NEJMoa2106596) and transduction efficency by single cell RNA sequencing (scRNAseq) using commercial serum-free media in the presence of the epigenetic modifier UM171 and early acting cytokines (DP: refers to 10.1056/NEJMoa2106596; for cultured samples at day4 and day 7: SFT6: stem cell factor, 100ng/mL; flt3 ligand, 50ng/mL). Cells of DP and its expanded samples were transduced with a lentiviral vector expressing the IDUA gene. WPRE transcript quantification was used as surrogate of LV IDUA expression to evaluated transduction efficiency.2026/06/10GSE292605GSM8862354GSM8862356GSM8862355GSM886235724676292605Homo sapiens10.1056/NEJMoa2106596