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single cell transcriptome analysis of regulated transgenic GATA1 expression as a gene therapy for Diamond-Blackfan anemia

GSE261450 Homo sapiens Expression profiling by high throughput sequencing 4 samples Submitted 2024/10/14 Platform GPL24676
Summary
We analyzed the transcriptional effects of transgenic expression of our erythroid-specific codon-optimized GATA1 lentiviral vector in differentiating red blood cells of patients diagnosed with Diamond-Blackfan anemia.
Published in
Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia
Voit RA, Liao X, Caulier A et al. · Cell stem cell 2025 · PMID 39532107 · doi:10.1016/j.stem.2024.10.012
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Also filed as BioProject PRJNA1086971 and SRA study SRP494878. Searching any of these in the dataset finder brings you back here.

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