Article
Targeted genome editing in human repopulating haematopoietic stem cells.
Nature - 12 Jun 2014
Genovese Pietro, Schiroli Giulia, Escobar Giulia, Tomaso Tiziano Di, Firrito Claudia, Calabria Andrea, Moi Davide, Mazzieri Roberta, Bonini Chiara, Holmes Michael C, Gregory Philip D, van der Burg Mirjam, Gentner Bernhard, Montini Eugenio, Lombardo Angelo, Naldini Luigi
Abstract excerpt
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...
Topics
- Animals
- Antigens, CD34
- DNA, Complementary
- Endonucleases
- Fetal Blood
- Gene Targeting
- Genome, Human
- Hematopoiesis
- Hematopoietic Stem Cell Transplantation
- Hematopoietic Stem Cells