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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Bibliografiske detaljer
Main Authors: Anguela, Xavier M., Sharma, Rajiv, Doyon, Yannick, Miller, Jeffrey C., Li, Hojun, Haurigot, Virginia, Rohde, Michelle E., Wong, Sunnie Y., Davidson, Robert J., Zhou, Shangzhen, Gregory, Philip D., Holmes, Michael C., High, Katherine A.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2013
Fag:
Gene Therapy
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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