DISCOVER-seq in relation to allele-specific correction as a therapy for GATA2 deficency
This study aims to investigate the potential use of gene editing as a therapy for the disease GATA2 deficiency. GATA2 deficiency is a disease caused by dysfunction of the hematopoietic stem cells. Therefore, the study focuses on the editing of GATA2 in hematopoietic stem cells. The study utilizes CRISPR/Cas9 to induce targeted DSBs and uses rAAV6 as a template for HDR. As a part of the study, the high-throughput sequencing-based method DISCOVER-seq has been used to nominate off-targets for the sgRNA used in the study.