Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

Schiroli, Giulia; Conti, Anastasia; Ferrari, Samuele; della Volpe, Lucrezia; Jacob, Aurelien; Albano, Luisa; Beretta, Stefano; Calabria, Andrea; Vavassori, Valentina; Gasparini, Patrizia; Salataj, Eralda; Ndiaye-Lobry, Delphine; Brombin, Chiara; Chaumeil, Julie; Montini, Eugenio; Merelli, Ivan; Genovese, Pietro; Naldini, Luigi; Di Micco, Raffaella

doi:10.1016/j.stem.2019.02.019

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

Summary Precise gene editing in hematopoietic stem and progenitor cells (HSPCs) holds promise for treating genetic diseases. However, responses triggered by programmable nucleases in HSPCs are poorly characterized and may negatively impact HSPC engraftment and long-term repopulation capacity. Her…