Introducing a G-Makassar Variant in HSCs by In Vivo Base Editing Treats Sickle Cell Disease in Mice

Investigators demonstrated successful production of helper-dependent adenoviral vectors expressing an all-in-one base editor mapping to the sickle mutation site. In HSC-enriched cells from sickle cell disease patients, transduction with the base editing vector in vitro resulted in 35% GTG to GCG conversion and phenotypic improvements of derived red blood cells.