In vivo HSC prime editing rescues sickle cell disease in a mouse model
Sign Up to like & getrecommendations! Published in 2023 at "Blood"
DOI: 10.1182/blood.2022018252
Abstract: Key Points • Correction of the sickle-cell mutation and disease phenotypes is achieved by in vivo HSC transduction with vectorized prime editors.• Our approach for in vivo HSC prime editing that does not require HSC… read more here.
Keywords: disease; prime editing; vivo hsc; hsc prime ... See more keywords