Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing
M. Mietzsch, A. R. Nelson, J. Hsi, J. Zachary, L. Potts, P. Chipman, M. Ghanem, N. Khandekar, I. E. Alexander, G. J. Logan, J. T. Huiskonen, R. McKenna
This study uses cryo-EM to analyze antibodies against AAV9 vectors, identifying residues that enable capsid engineering to avoid neutralization and allow redosing.
bioRxiv · May 1, 2024 · PreprintImmunology