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 language | English (US) |
|---|---|
| Pages (from-to) | 1124-1131 |
| Number of pages | 8 |
| Journal | Human gene therapy |
| Volume | 31 |
| Issue number | 19-20 |
| DOIs | |
| State | Published - Oct 2020 |
Bibliographical note
Publisher Copyright:© Karina Krotova and George Aslanidi 2020; Published by Mary Ann.
Keywords
- Adeno-associated virus
- Assay development
- Neutralizing antibodies
- Selective inhibitor of ampk