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AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye.

ABSTRACT: Ocular gene therapy has evolved rapidly into the clinical realm due to promising pre-clinical proof-of-concept studies, recognition of the high unmet medical need of blinding disorders, and the excellent safety profile of the most commonly used vector system, the adeno-associated virus (AAV). With several trials exposing subjects to AAV, investigators independently report about cases with clinically evident inflammation in treated eyes despite the concept of ocular immune privilege. Here, we provide a detailed analysis of innate and adaptive immune response to clinical-grade AAV8 in non-human primates and compare this to preliminary clinical data from a retinal gene therapy trial for CNGA3-based achromatopsia (ClinicalTrials.gov: 02610582).

SUBMITTER: Reichel FF 

PROVIDER: S-EPMC5768589 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature

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AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye.

Reichel Felix F FF   Dauletbekov Daniyar L DL   Klein Reinhild R   Peters Tobias T   Ochakovski G Alex GA   Seitz Immanuel P IP   Wilhelm Barbara B   Ueffing Marius M   Biel Martin M   Wissinger Bernd B   Michalakis Stylianos S   Bartz-Schmidt Karl Ulrich KU   Fischer M Dominik MD  

Molecular therapy : the journal of the American Society of Gene Therapy 20170831 12

Ocular gene therapy has evolved rapidly into the clinical realm due to promising pre-clinical proof-of-concept studies, recognition of the high unmet medical need of blinding disorders, and the excellent safety profile of the most commonly used vector system, the adeno-associated virus (AAV). With several trials exposing subjects to AAV, investigators independently report about cases with clinically evident inflammation in treated eyes despite the concept of ocular immune privilege. Here, we pro  ...[more]

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