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Review
. 2015 Jun;15(6):845-55.
doi: 10.1517/14712598.2015.1035645.

Strategies to circumvent humoral immunity to adeno-associated viral vectors

Longping V Tse  1 , Sven Moller-Tank, Aravind Asokan
Affiliations
Review

Strategies to circumvent humoral immunity to adeno-associated viral vectors

Longping V Tse et al. Expert Opin Biol Ther. 2015 Jun.

Abstract

Introduction: Recent success in gene therapy of certain monogenic diseases in the clinic has infused enthusiasm into the continued development of recombinant adeno-associated viral (AAV) vectors as next-generation biologics. However, progress in clinical trials has also highlighted the challenges posed by the host humoral immune response to AAV vectors. Specifically, while pre-existing neutralizing antibodies (NAbs) limit the cohort of eligible patients, NAb generation following treatment prevents vector re-dosing.

Areas covered: In this review, we discuss a spectrum of complementary strategies that can help circumvent the host humoral immune response to AAV.

Expert opinion: Specifically, we present a dual perspective, that is, vector versus host, and highlight the clinical attributes, potential caveats and limitations as well as complementarity associated with the various approaches.

Keywords: adeno-associated virus; capsid; humoral immune response; neutralizing antibody; recombinant adeno-associated viral vectors.

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