Cell-Based Determination of Neutralizing Antibodies Against Adeno-Associated Virus in Cardiac Gene Therapy

Anjali J. Ravichandran, Renata Mazurek, Kiyotake Ishikawa

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

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.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages293-304
Number of pages12
DOIs
StatePublished - 2022

Publication series

NameMethods in Molecular Biology
Volume2573
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029

Keywords

  • Adeno-associated virus (AAV)
  • Cardiac gene therapy
  • Luciferase assay
  • Neutralizing antibody assay
  • Neutralizing antibody titer

Fingerprint

Dive into the research topics of 'Cell-Based Determination of Neutralizing Antibodies Against Adeno-Associated Virus in Cardiac Gene Therapy'. Together they form a unique fingerprint.

Cite this