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Robust ZFN-mediated genome editing in adult hemophilic mice
Monogenic diseases, including hemophilia, represent ideal targets for genome-editing approaches aimed at correcting a defective gene. Here we report that systemic adeno-associated virus (AAV) vector delivery of zinc finger nucleases (ZFNs) and corrective donor template to the predominantly quiescent...
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| Main Authors: | , , , , , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
American Society of Hematology
2013
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3821724/ https://ncbi.nlm.nih.gov/pubmed/24085764 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2013-04-497354 |
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