Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy

• Published in: Molecular therapy Vol. 31; no. 7; pp. 1979 - 1993
• Main Authors: Logan, Grant J., Mietzsch, Mario, Khandekar, Neeta, D’Silva, Arlene, Anderson, Daniel, Mandwie, Mawj, Hsi, Jane, Nelson, Austin R., Chipman, Paul, Jackson, Jennifer, Schofield, Peter, Christ, Daniel, Goodnow, Christopher C., Reed, Joanne H., Farrar, Michelle A., McKenna, Robert, Alexander, Ian E.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 05.07.2023; American Society of Gene & Cell Therapy
• Subjects: adeno-associated virus vector; affinity; Animals; anti-vector immunity; Antibodies, Monoclonal - genetics; Antibodies, Monoclonal - therapeutic use; capsid; Capsid - chemistry; Capsid Proteins - chemistry; capsid structure; Cryoelectron Microscopy; Dependovirus; Genetic Therapy; Genetic Vectors - genetics; Humans; Infant; Mice; monoclonal antibody; neutralization; Original; recombinant AAV; spinal muscular atrophy; Zolgensma; capsid structure; neutralization; Zolgensma; capsid; recombinant AAV; spinal muscular atrophy; monoclonal antibody; anti-vector immunity; adeno-associated virus vector; affinity

Success in the treatment of infants with spinal muscular atrophy (SMA) underscores the potential of vectors based on adeno-associated virus (AAV). However, a major obstacle to the full realization of this potential is pre-existing natural and therapy-induced anti-capsid humoral immunity. Structure-g...