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Expert Opin Biol Ther. 2015 Jun;15(6):845-55. doi: 10.1517/14712598.2015.1035645.

Strategies to circumvent humoral immunity to adeno-associated viral vectors.

Author information

1
University of North Carolina, Gene Therapy Center , CB#7352, Thurston Building, Chapel Hill, NC 27599 , USA.

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

PMID:
25985812
PMCID:
PMC4689135
DOI:
10.1517/14712598.2015.1035645
[Indexed for MEDLINE]
Free PMC Article

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