Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy

Vedell Louis Jeune, Jakob A. Joergensen, Roger J. Hajjar, Thomas Weber

Research output: Contribution to journalReview articlepeer-review

226 Scopus citations

Abstract

Adeno-associated virus (AAV)-based vectors are promising tools for gene therapeutic applications, in part because AAVs are nonpathogenic viruses, and vectors derived from them can drive long-term transgene expression without integration of the vector DNA into the host genome. AAVs are not strongly immunogenic, but they can, nonetheless, give rise to both a cellular and humoral immune response. As a result, a significant fraction of potential patients for AAV-based gene therapy harbors pre-existing antibodies against AAV. Because even very low levels of antibodies can prevent successful transduction, antecedent anti-AAV antibodies pose a serious obstacle to the universal application of AAV gene therapy. In this review, we discuss the current knowledge of the role of anti-AAV antibodies in AAV-based gene therapy with a particular emphasis on approaches to overcome the hurdle that they pose.

Original languageEnglish
Pages (from-to)59-67
Number of pages9
JournalHuman gene therapy methods
Volume24
Issue number2
DOIs
StatePublished - 1 Apr 2013

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