Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.

ABSTRACT: The ability to effectively deliver genetic material to vascular endothelial cells remains one of the greatest unmet challenges facing the development of gene therapies to prevent diseases with underlying vascular etiology, such as diabetes, atherosclerosis, and age-related macular degeneration. Herein, we assess the effectiveness of an rAAV2-based capsid mutant vector (Y272F, Y444F, Y500F, Y730F, T491V; termed QuadYF+TV) with strong endothelial cell tropism at transducing the vasculature after systemic administration. Intravenous injection of QuadYF+TV resulted in widespread transduction throughout the vasculature of several major organ systems, as assessed by in vivo bioluminescence imaging and postmortem histology. Robust transduction of lung tissue was observed in QuadYF+TV-injected mic

SUBMITTER: Lipinski DM 

PROVIDER: S-EPMC4651034 | biostudies-literature | 2015 Nov

REPOSITORIES: biostudies-literature