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Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements

20 Feb 2024

Abstract excerpt

Recombinant adeno-associated virus (AAV) vectors are the leading delivery vehicle used for in vivo gene therapies. Anti-AAV antibodies (AAV Abs) can interact with the viral capsid component of an AAV-based gene therapy (GT). Therefore, patients with preexisting AAV Abs (seropositive patients) are often excluded from GT trials to prevent treatment of patients who are unlikely to benefit 1 or may have a higher risk...

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PMID: 38496304
DOI: 10.1016/j.omtm.2024.101217

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