Abstract
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 language | English (US) |
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Article number | 523 |
Journal | Biomedicines |
Volume | 11 |
Issue number | 2 |
DOIs | |
State | Published - Feb 2023 |
Bibliographical note
Funding Information:This project was supported by NIH R01HL131586 and FDA/CEBR FD007483 grants.
Publisher Copyright:
© 2023 by the authors.
Keywords
- adeno-associated virus
- assay development
- patient population
- preclinical animal model
- serotype-specific neutralizing antibodies
PubMed: MeSH publication types
- Journal Article