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

V Louis Jeune, JA Joergensen, RJ Hajjar… - Human gene therapy …, 2013 - liebertpub.com
V Louis Jeune, JA Joergensen, RJ Hajjar, T Weber
Human gene therapy methods, 2013liebertpub.com
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 …
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.
Mary Ann Liebert