Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [scRNA]
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ABSTRACT: To determine the heterogeneity of fetal hemoglobin induction at a single-cell level, CD235a+ erythroid cells were extracted from mouse BM after 17 weeks xenotransplantation. Single-cell RNA-sequencing (10x Genomics) showed 82% of the cells more than 30% g-globin expression in edited cells compared to unedited controls (26.7%).
ORGANISM(S): Homo sapiens
PROVIDER: GSE275063 | GEO | 2024/08/21
REPOSITORIES: GEO
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