CRISPR/Cas 9 mediated targeting of MIR155HG in primary human T cells creates genomic deletions that disrupt transcription of mature microRNA-155 (miR-155, a miR associated with inflammation). Applying this treatment to donor T cells prior to allogenic hematopoietic stem cell transplantation (allo-HSCT) when treating hematological malignancies and other primary bone marrow disorders can prevent the development of aGVHD in patients.
More than 8,000 patients receive allo-HSCT annually in the US alone as a cure for hematologic malignancies and other primary bone marrow disorders. However, the major barrier for the success of allo-HSCT is the high incidence of aGVHD and its associated morbidity and mortality. Acute GVHD…