Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing
Mario Mietzsch, Jane Hsi, Austin R. Nelson, Neeta Khandekar, Ann-Maree Huang, Nicholas J. C. Smith, Jon Zachary, Lindsay Potts, Michelle Anne Farrar, Paul R. Chipman, Mohammad Ghanem, Ian E. Alexander, Grant J. Logan, Juha T. Huiskonen, ROBERT V. McKENNA
This study uses cryo-EM to analyze antibodies against AAV9 vectors, identifying residues that enable capsid engineering to avoid neutralization and allow redosing.
Nature Communications · Apr 19, 2025Peer-reviewedImmunology