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...

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Bibliographische Detailangaben
Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: Holmgaard, Andreas, Askou, Anne Louise, Benckendorff, Josephine Natalia Esther, Thomsen, Emil Aagaard, Cai, Yujia, Bek, Toke, Mikkelsen, Jacob Giehm, Corydon, Thomas J.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2017
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5626917/
https://ncbi.nlm.nih.gov/pubmed/29246327
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.08.016
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