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Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts

19 Apr 2025

Abstract excerpt

Monoclonal antibodies are useful tools to dissect the neutralizing antibody response against the adeno-associated virus (AAV) capsids that are used as gene therapy delivery vectors. The presence of pre-existing neutralizing antibodies in large portions of the human population poses a significant challenge for AAV-mediated gene therapy, primarily targeting the capsid leading to vector inactivation and loss of...

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PMID: 40253479
DOI: 10.1038/s41467-025-59088-4

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