Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid
Recchia Martino, Qiang Wang, Hao Xu, Gui Hu, Peter Bell, Edgardo J. Arroyo, Joshua J. Sims, James M. Wilson
Abstract
, would be useful to capsid engineers as they develop AAV vectors for gene therapy applications and characterize their interactions with native or engineered receptors. Here, we use the AAV-PHP.B-Ly6a model system to assess the impact of receptor affinity on the systemic delivery and endothelial penetration properties of AAV-PHP.B vectors. We discuss how receptor affinity analysis can be used to isolate vectors with optimized properties, improve the interpretation of library selections, and ultimately translate vector activities between preclinical animal models and humans.
Topics & Concepts
CapsidTransduction (biophysics)BiologyReceptorComputational biologyGene deliveryCell biologyIn vivoGenetic enhancementGeneGeneticsBiophysicsVirus-based gene therapy researchRNA Interference and Gene DeliveryCRISPR and Genetic Engineering