Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

Targeted gene editing in hematopoietic stem cells (HSCs) is a promising treatment for several diseases. However, the limited efficiency of homology-directed repair (HDR) in HSCs and the unknown impact of the procedure on clonal composition and dynamics upon transplantation have hampered clinical tra...

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Udgivet i:Nat Biotechnol
Main Authors: Ferrari, Samuele, Jacob, Aurelien, Beretta, Stefano, Unali, Giulia, Albano, Luisa, Vavassori, Valentina, Cittaro, Davide, Lazarevic, Dejan, Brombin, Chiara, Cugnata, Federica, Kajaste-Rudnitski, Anna, Merelli, Ivan, Genovese, Pietro, Naldini, Luigi
Format: Artigo
Sprog:Inglês
Udgivet: 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7610558/
https://ncbi.nlm.nih.gov/pubmed/32601433
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41587-020-0551-y
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