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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [scRNA]

GSE275063 Homo sapiens Expression profiling by high throughput sequencing 6 samples Submitted 2024/08/21 Platform GPL24676
Summary
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%).
Published in
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34(+) cell therapy to induce fetal hemoglobin for sickle cell disease
Katta V, O'Keefe K, Li Y et al. · Molecular therapy : the journal of the American Society of Gene Therapy 2024 · PMID 39086133 · doi:10.1016/j.ymthe.2024.07.022
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Also filed as BioProject PRJNA1149034 and SRA study SRP526907. Searching any of these in the dataset finder brings you back here.

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