Single-cell profiling of a clinically applicable lentiviral vector for the treatment of Diamond-Blackfan anemia. Single-cell profiling of a clinically applicable lentiviral vector for the treatment of Diamond-Blackfan anemia

We applied single-cell RNA sequencing analysis to study the transcriptomic landscapes of a clinically applicable lentiviral vector in RPS19-deficient CD34+ hematopoietic stem and progenitor cells Overall design: Single cell transcriptional profiling using the 10x Genomics platform was performed on CRISPR edited RPS19-dedicient CD34+ hematopoietic stem and progenitor cells with or without treatment of lentiviral therapeutic vector. The cells were generated using FACS isolated from 2 RPS19-deficient samples, 2 vector treated samples, and 1 normal control. Submitter declares that the raw data will be deposited in dbGaP due to patient privacy concerns.