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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Autors principals: 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
Idioma:Inglês
Publicat: American Society of Hematology 2013
Matèries:
Gene Therapy
Accés en línia: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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