Ontology highlight
ABSTRACT:
SUBMITTER: Holmgaard A
PROVIDER: S-EPMC5626917 | biostudies-literature | 2017 Dec
REPOSITORIES: biostudies-literature
Holmgaard Andreas A Askou Anne Louise AL Benckendorff Josephine Natalia Esther JNE Thomsen Emil Aagaard EA Cai Yujia Y Bek Toke T Mikkelsen Jacob Giehm JG Corydon Thomas J TJ
Molecular therapy. Nucleic acids 20170921
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 vivo to selectively ablate the vascular endothelial growth factor A (Vegfa) gene in mice. By generating LVs encoding SpCas9 targeted to Vegfa, and in parallel the fluorescent eGFP marker protein, we ...[more]