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

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%). Single-cell RNA-sequencing analysis of CD235a+ cells from transplanted NBSGW mice bone marrow (BM) derived from three SCD donors at 17 weeks.