Preclinical development of DBAS gene therapy

We developed a novel self-inactivating third-generation lentiviral vector encoding RPS19 for Diamond-Blackfan anemia syndrome (DBAS) gene therapy. We optimized the LV design, improved transduction efficiency and assess potential therapeutic efficacy in this study using a novel, biologically relevant, human cellular model of DBAS based on heterozygous disruption of RPS19 in healthy donor CD34+ hematopoietic stem/progenitor cells.