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Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells

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 Hematopoietic Stem Cells (HSCs) has remained elusive. Here we show that poor permissive...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4082311/
https://www.ncbi.nlm.nih.gov/pubmed/24870228
http://dx.doi.org/10.1038/nature13420