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

• Main Authors: Andreas Holmgaard (Author), Anne Louise Askou (Author), Josephine Natalia Esther Benckendorff (Author), Emil Aagaard Thomsen (Author), Yujia Cai (Author), Toke Bek (Author), Jacob Giehm Mikkelsen (Author), Thomas J. Corydon (Author)
• Formato: Livro
• Publicado em: Elsevier, 2017-12-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...