HDAd dual prime editor

HDAd dual prime editor is an in vivo gene therapy platform utilizing a helper-dependent adenoviral (HDAd) vector to deliver a multiplexed prime editing system. Developed by researchers at the University of Washington and the Broad Institute, this system is designed to treat hemoglobinopathies, specifically sickle cell disease. It employs a dual prime editing strategy to simultaneously introduce a naturally occurring, non-pathological mutation in the erythropoietin receptor (EPOR) gene—creating a truncated EPOR (tEPOR) that confers hypersensitivity to erythropoietin—and correct the pathogenic mutation in the β-globin gene (HBB). The tEPOR modification is intended to allow for the autonomous expansion of gene-corrected erythroid cells, potentially eliminating the need for toxic chemotherapy-based selection during hematopoietic stem and progenitor cell (HSPC) gene therapy.

Other names

HDAd-mediated dual prime editorHDAd5/35 dual prime editor