Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid

Author:

Martino R. Alexander1,Wang Qiang1,Xu Hao1,Hu Gui1,Bell Peter1,Arroyo Edgardo J.1,Sims Joshua J.1,Wilson James M.1ORCID

Affiliation:

1. Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA

Abstract

Novel methods for measuring adeno-associated virus (AAV)-receptor affinities, especially in relation to vector performance in vivo , 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.

Funder

Amicus Therapeutics

Passage Bio

Publisher

American Society for Microbiology

Subject

Virology,Insect Science,Immunology,Microbiology

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