Ontology highlight
ABSTRACT:
SUBMITTER: Genovese P
PROVIDER: S-EPMC4082311 | biostudies-literature | 2014 Jun
REPOSITORIES: biostudies-literature
Genovese Pietro P Schiroli Giulia G Escobar Giulia G Tomaso Tiziano Di TD Firrito Claudia C Calabria Andrea A Moi Davide D Mazzieri Roberta R Bonini Chiara C Holmes Michael C MC Gregory Philip D PD van der Burg Mirjam M Gentner Bernhard B Montini Eugenio E Lombardo Angelo A Naldini Luigi L
Nature 20140528 7504
Targeted genome editing by artificial nucleases has brought the goal of site-specific transgene integration and gene correction within the reach of gene therapy. However, its application to long-term repopulating haematopoietic stem cells (HSCs) has remained elusive. Here we show that poor permissiveness to gene transfer and limited proficiency of the homology-directed DNA repair pathway constrain gene targeting in human HSCs. By tailoring delivery platforms and culture conditions we overcame th ...[more]