AI Article Synopsis
- * It highlights the importance of screening patients for neutralizing antibodies that can inhibit AAV delivery, which is crucial for treatment success.
- * A cell-based neutralizing antibody assay, combined with a luciferase assay, is detailed as a method to measure existing patient antibodies that may affect AAV gene therapy outcomes.
Article Abstract
The field of cardiac gene therapy has seen the rising use of adeno-associated viral (AAV) vectors as a promising therapeutic option for cardiac diseases and heart failure. To achieve intended results of AAV delivery, a majority of clinical studies screen patients for existing neutralizing antibodies that could inhibit the effects of the administered AAV and confound treatment efficacy. The cell-based neutralizing antibody assay offers a method of quantifying and identifying a patient's existing neutralizing antibodies against specific serotypes. Combined with the luciferase assay, the neutralizing antibody assay tests the ability of patient antibodies in the blood to prevent gene transduction of AAV-encoded luciferase gene at ranging serial dilutions. This chapter provides a protocol and experimental techniques to determine the presence of neutralizing antibodies against AAV in the blood.
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| http://dx.doi.org/10.1007/978-1-0716-2707-5_22 | DOI Listing |
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