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In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells
Virus-based gene therapy by CRISPR/Cas9-mediated genome editing and knockout may provide a new option for treatment of inherited and acquired ocular diseases of the retina. In support of this notion, we show that Streptococcus pyogenes (Sp) Cas9, delivered by lentiviral vectors (LVs), can be used in...
Autores principales: | Holmgaard, Andreas, Askou, Anne Louise, Benckendorff, Josephine Natalia Esther, Thomsen, Emil Aagaard, Cai, Yujia, Bek, Toke, Mikkelsen, Jacob Giehm, Corydon, Thomas J. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5626917/ https://www.ncbi.nlm.nih.gov/pubmed/29246327 http://dx.doi.org/10.1016/j.omtn.2017.08.016 |
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