In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells

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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Bibliographic Details
Main Authors: Andreas Holmgaard, Anne Louise Askou, Josephine Natalia Esther Benckendorff, Emil Aagaard Thomsen, Yujia Cai, Toke Bek, Jacob Giehm Mikkelsen, Thomas J. Corydon
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
SpCas9
lentiviral vector
retinal gene knockout
RPE cells
VEGFA
subretinal delivery
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117302445

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http://www.sciencedirect.com/science/article/pii/S2162253117302445

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