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Clonal analysis and transcriptional profiling of ex vivo expanded mobilized peripheral blood hematopoietic stem and progenitor [MPS_scRNAseq]

ABSTRACT: 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.

ORGANISM(S): Homo sapiens

PROVIDER: GSE292604 | GEO | 2026/06/10

REPOSITORIES: GEO

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Clonal analysis and transcriptional profiling of ex vivo expanded mobilized peripheral blood hematopoietic stem and progenitor [bulkRNAseq]

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