{"database":"GEO","file_versions":[{"headers":{"Content-Type":["application/json"]},"body":{"files":{"Other":["ftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE292nnn/GSE292604/"]},"type":"primary"},"statusCode":"OK","statusCodeValue":200}],"scores":null,"additional":{"omics_type":["Transcriptomics"],"species":["Homo sapiens"],"gds_type":["Expression profiling by high throughput sequencing"],"full_dataset_link":["https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE292604"],"repository":["GEO"],"entry_type":["GSE"],"additional_accession":[]},"is_claimable":false,"name":"Clonal analysis and transcriptional profiling of ex vivo expanded mobilized peripheral blood hematopoietic stem and progenitor [MPS_scRNAseq]","description":"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.","dates":{"publication":"2026/06/10"},"accession":"GSE292604","cross_references":{"GSM":["GSM8862353","GSM8862350","GSM8862352","GSM8862351","GSM8862349","GSM8862348"],"GPL":["24676"],"GSE":["292604"],"taxon":["Homo sapiens"],"DOI":["10.1056/NEJMoa2106596"]}}