Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal Models and Humans

Hisae Kuoch, Karina Krotova, Melanie L. Graham, Mark L. Brantly, George Aslanidi

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


The accurate assessment of AAV-specific pre-existing humoral immunity due to natural viral infection is critical for the efficient use of clinical gene therapy. The method described in the present study applies equivalent infection conditions to each AAV serotype (AAV1, AAV2, AAV3, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, and AAVAnc80L65). In the current study, we validated the assay by assessing AAV-neutralizing antibody titers in a limited cohort of random human donors and well-established preclinical large animal models, including dogs and non-human primates (NHPs). We achieved a rapid and accurate evaluation of neutralizing titers for each individual subject that can be used for clinical enrollment based on specific AAV serotypes and individualized selection of the most suitable AAV serotype for each specific patient.

Original languageEnglish (US)
Article number523
Issue number2
StatePublished - Feb 2023

Bibliographical note

Funding Information:
This project was supported by NIH R01HL131586 and FDA/CEBR FD007483 grants.

Publisher Copyright:
© 2023 by the authors.


  • adeno-associated virus
  • assay development
  • patient population
  • preclinical animal model
  • serotype-specific neutralizing antibodies

PubMed: MeSH publication types

  • Journal Article


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