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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Detalhes bibliográficos
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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2013
Assuntos:
Gene Therapy
Acesso em linha: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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