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Molecular Therapy : the Journal of the American Society of Gene Therapy|April 4, 2023
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapyGrant J Logan, Mario Mietzsch, Neeta Khandekar, et al.
JCI Insight|July 9, 2025
Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritisAnne Cao Le, Virginie Oudin, Jonathan Dick, et al.
Proceedings of the National Academy of Sciences of the United States of America|March 23, 2021
Gain-of-function factor H-related 5 protein impairs glomerular complement regulation resulting in kidney damageTalat H Malik, Daniel P Gitterman, Deborah P Lavin, et al.
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