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

Reichel, Felix F.; Dauletbekov, Daniyar L.; Klein, Reinhild; Peters, Tobias; Ochakoyski, G. Alex; Seitz, Immanuel P.; Wilhelm, Barbara; Üffing, Marius; Biel, Martin; Wissinger, Bernd; Michalakis, Stylianos; Bartz-Schmidt, Karl Ulrich und Fischer, M. Dominik (2017): AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye. In: Molecular therapy, Bd. 25, Nr. 12: S. 2648-2660

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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Chemie und Pharmazie > Department für Pharmazie - Zentrum für Pharmaforschung
• Fakultätsübergreifende Einrichtungen: Center for Integrated Protein Science Munich (CIPSM)
• Themengebiete: 500 Naturwissenschaften und Mathematik > 540 Chemie
• ISSN: 1525-0016
• Sprache: Englisch