Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay

Karina Krotova, George Aslanidi

Research output: Contribution to journalArticlepeer-review

Abstract

Adeno-associated virus (AAV)-based gene therapy is undergoing major expansion into clinical practice, with two treatments currently being granted Food and Drug Administration (FDA) approval. However, the presence of preexisting neutralizing antibodies (NAB) is one of the significant hurdles for the clinical application of AAV vectors that significantly limits the patient population, which benefits from the treatment. A reliable diagnostic to evaluate the patient's seropositivity is required to ensure the effectiveness of the AAV-mediated therapeutic. Here, we describe a simple method for the determination of AAV NAB activity based on our finding that Compound C makes HEK293 cell highly permissive for infection by 10 commonly used AAV serotypes.

Original languageEnglish (US)
Pages (from-to)1124-1131
Number of pages8
JournalHuman gene therapy
Volume31
Issue number19-20
DOIs
StatePublished - Oct 2020

Bibliographical note

Funding Information:
This project was supported by NIH/NIGM 1R01HL131586 and startup funds from the Hormel Institute.

Publisher Copyright:
© Karina Krotova and George Aslanidi 2020; Published by Mary Ann.

Keywords

  • Adeno-associated virus
  • Assay development
  • Neutralizing antibodies
  • Selective inhibitor of ampk

PubMed: MeSH publication types

  • Journal Article
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

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