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

ABSTRACT: 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.

ORGANISM(S): Homo sapiens

PROVIDER: GSE261450 | GEO | 2024/10/14

REPOSITORIES: GEO