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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2014
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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 |
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