Article
Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.
Human gene therapy - 1 Nov 2015
Lipinski Daniel M, Reid Chris A, Boye Sanford L, Peterson James J, Qi Xiaoping, Boye Shannon E, Boulton Michael E, Hauswirth William W
Abstract excerpt
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...
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